21st Century Addison's Disease Sourcebook: Clinical Data for Patients, Families, and Physicians, including Adrenal Insufficiency, Adrenocortical Hypofunction, Hypocortisolism, and Related Conditions
Edition 1.0 / July 2011
Medical Ventures Press, National Institutes of Health
Smashwords Edition
Copyright 2011 Progressive Management
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IMPORTANT NOTE: Information in this e-book is not a substitute for professional medical advice. If you have or suspect that you have any illness, you must consult with a physician or professional healthcare provider! Call 911 and get to the nearest emergency room if you have serious or worsening symptoms.
This material represents a snapshot in time, with authoritative information formatted for ebook reading that was up-to-date at the moment of publication.
For the latest updates, please be sure to visit the trustworthy websites found in our Guide to Leading Medical Websites (Section B).
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TABLE OF CONTENTS
SECTION A: Addison's Disease and Related Conditions
SECTION B: Guide to Leading Medical Websites
SECTION C: Medical Fundamentals - Mini-Encyclopedia
SECTION D: Cancer Fundamentals - Mini-Encyclopedia
SECTION E: Affordable Care Act (ACA)
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SECTION A: ADDISON'S DISEASE AND RELATED CONDITIONS
Addison's Disease
Also called: Adrenal insufficiency, Adrenocortical hypofunction, Hypocortisolism
The primary NIH organization for research on Addison's Disease is the National Institute of Diabetes and Digestive and Kidney Diseases
Your adrenal glands are just above your kidneys. The outside layer of these glands makes hormones that help your body respond to stress and regulate your blood pressure and water and salt balance. Addison's disease occurs if the adrenal glands don't make enough of these hormones.
A problem with your immune system usually causes Addison's disease. The immune system mistakenly attacks your own tissues, damaging your adrenal glands.
Symptoms include
Weight loss
Muscle weakness
Fatigue that gets worse over time
Low blood pressure
Patchy or dark skin
Lab tests can confirm that you have Addison's disease. If you don't treat it, it can be fatal. You will need to take hormone pills for the rest of your life. If you have Addison's disease, you should carry an emergency ID. It should say that you have the disease, list your medicines and say how much you need in an emergency.
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Adrenal Insufficiency and Addison's Disease
What is adrenal insufficiency? What do adrenal hormones do? What are the symptoms of adrenal insufficiency? What causes Addison's disease? What causes secondary adrenal insufficiency? How is adrenal insufficiency diagnosed? How is adrenal insufficiency treated? What special problems can occur with adrenal insufficiency? How can someone with adrenal insufficiency prepare for an emergency
What is adrenal insufficiency?
Adrenal insufficiency is an endocrine-or hormonal-disorder that occurs when the adrenal glands do not produce enough of certain hormones. The adrenal glands are located just above the kidneys. Adrenal insufficiency can be primary or secondary.
Primary adrenal insufficiency, also called Addison's disease, occurs when the adrenal glands are damaged and cannot produce enough of the hormone cortisol and often the hormone aldosterone. Addison's disease affects one to four of every 100,000 people, in all age groups and both sexes. (Munver R, Volfson IA. Adrenal insufficiency: diagnosis and management. Current Urology Reports. 2006;7:80–85.)
Secondary adrenal insufficiency occurs when the pituitary gland-a bean-sized organ in the brain-fails to produce enough adrenocorticotropin (ACTH), a hormone that stimulates the adrenal glands to produce cortisol. If ACTH output is too low, cortisol production drops. Eventually, the adrenal glands can shrink due to lack of ACTH stimulation. Secondary adrenal insufficiency is much more common than Addison's disease.
What do adrenal hormones do?
Cortisol
Cortisol belongs to a class of hormones called glucocorticoids, which affect almost every organ and tissue in the body. Cortisol's most important job is to help the body respond to stress. Among its many vital tasks, cortisol helps
maintain blood pressure and cardiovascular function
slow the immune system's inflammatory response
maintain levels of glucose-a form of sugar used for energy-in the blood
regulate the metabolism of proteins, carbohydrates, and fats
The amount of cortisol produced by the adrenals is precisely balanced. Like many other hormones, cortisol is regulated by the brain's hypothalamus and the pituitary gland. First, the hypothalamus releases a "trigger" hormone called corticotropin-releasing hormone (CRH) that signals the pituitary gland. The pituitary responds by sending out ACTH, which in turn stimulates the adrenal glands. The adrenal glands respond by producing cortisol. Completing the cycle, cortisol then signals back to both the pituitary and hypothalamus to decrease these trigger hormones.
The hypothalamus sends CRH to the pituitary, which responds by sending out ACTH. ACTH then causes the adrenals to release cortisol into the bloodstream.
Aldosterone
Aldosterone belongs to a class of hormones called mineralocorticoids, also produced by the adrenal glands. Aldosterone helps maintain blood pressure and water and salt balance in the body by helping the kidneys retain sodium and excrete potassium. When aldosterone production falls too low, the kidneys are not able to regulate water and salt balance, leading to a drop in both blood volume and blood pressure.
What are the symptoms of adrenal insufficiency?
The symptoms of adrenal insufficiency usually begin gradually. The most common symptoms are
chronic, worsening fatigue
muscle weakness
loss of appetite
weight loss
Other symptoms can include
nausea
vomiting
diarrhea
low blood pressure that falls further when standing, causing dizziness or fainting
irritability and depression
a craving for salty foods due to salt loss
hypoglycemia, or low blood glucose
headache
sweating
in women, irregular or absent menstrual periods
Hyperpigmentation, or darkening of the skin, can occur in Addison's disease but not in secondary adrenal insufficiency. This darkening is most visible on scars; skin folds; pressure points such as the elbows, knees, knuckles, and toes; lips; and mucous membranes such as the lining of the cheek.
Because the symptoms progress slowly, they are often ignored until a stressful event like an illness or accident causes them to worsen. Sudden, severe worsening of symptoms is called an Addisonian crisis, or acute adrenal insufficiency. In most cases, symptoms of adrenal insufficiency become serious enough that people seek medical treatment before a crisis occurs. However, sometimes symptoms first appear during an Addisonian crisis.
Symptoms of an Addisonian or "adrenal" crisis include
sudden, penetrating pain in the lower back, abdomen, or legs
severe vomiting and diarrhea
dehydration
low blood pressure
loss of consciousness
If not treated, an Addisonian crisis can be fatal.
What causes Addison's disease?
Autoimmune Disorders
The gradual destruction of the adrenal cortex, the outer layer of the adrenal glands, by the body's immune system causes up to 80 percent of Addison's disease cases. In autoimmune disorders, the immune system makes antibodies that attack the body’s own tissues or organs and slowly destroy them. (Martorell PM, Roep BO, Smit JWA. Autoimmunity in Addison’s disease. The Netherlands Journal of Medicine. 2002;60(7):269-275)
Adrenal insufficiency occurs when at least 90 percent of the adrenal cortex has been destroyed. As a result, often both cortisol and aldosterone are lacking. Sometimes only the adrenal glands are affected. Sometimes other endocrine glands are affected as well, as in polyendocrine deficiency syndrome.
Polyendocrine deficiency syndrome is classified into two separate forms, type 1 and type 2. Type 1 is inherited and occurs in children. In addition to adrenal insufficiency, these children may have
underactive parathyroid glands, which produce a hormone that regulates calcium and phosphorus balance in the body
slow sexual development
pernicious anemia, a severe type of anemia
chronic candida infections, a type of fungal infection
chronic active hepatitis, a liver disease
Type 2, sometimes called Schmidt's syndrome, usually affects young adults and may include
an underactive thyroid gland, which produces hormones that regulate metabolism
slow sexual development
diabetes
vitiligo, a loss of pigment on areas of the skin
Scientists think type 2 polyendocrine deficiency syndrome is also inherited because often more than one family member has one or more endocrine deficiencies.
Tuberculosis
Tuberculosis (TB), an infection that can destroy the adrenal glands, accounts for less than 20 percent of cases of Addison's disease in developed countries.3 When adrenal insufficiency was first identified by Dr. Thomas Addison in 1849, TB was the most common cause of the disease. As TB treatment improved, the incidence of adrenal insufficiency due to TB of the adrenal glands greatly decreased. (Munver R, Volfson IA. Adrenal insufficiency: diagnosis and management. Current Urology Reports. 2006;7:80-85.)
Other Causes
Less common causes of Addison's disease are
chronic infection, mainly fungal infections
cancer cells spreading from other parts of the body to the adrenal glands
amyloidosis, a disease that causes abnormal protein buildup in, and damage to, various organs
surgical removal of the adrenal glands
AIDS-associated infections
bleeding into the adrenal glands
genetic defects including abnormal adrenal gland development, an inability of the adrenal gland to respond to ACTH, or a defect in adrenal hormone production
What causes secondary adrenal insufficiency?
Secondary adrenal insufficiency can be traced to a lack of ACTH. Without ACTH to stimulate the adrenal glands, the adrenals' production of cortisol drops. Aldosterone production is not usually affected.
A temporary form of secondary adrenal insufficiency may occur when a person who has been taking a synthetic glucocorticoid hormone such as prednisone for a long time stops taking the medication, either abruptly or gradually. Glucocorticoid hormones, which are often used to treat inflammatory illnesses such as rheumatoid arthritis, asthma, and ulcerative colitis, block the release of both CRH and ACTH. As a result, the adrenals may begin to atrophy—or shrink—from lack of ACTH stimulation and then fail to secrete sufficient levels of cortisol.
A person who stops taking a synthetic glucocorticoid hormone may have enough ACTH to function when healthy. However, when a person is under the stress of an illness, accident, or surgery, the person's body may not have enough ACTH to stimulate the adrenal glands to produce cortisol.
Another cause of secondary adrenal insufficiency is surgical removal of the noncancerous, ACTH-producing tumors of the pituitary gland that cause Cushing's disease. Cushing's disease is another disorder that leads to excess cortisol in the body. In this case, the source of ACTH is suddenly removed and replacement hormone must be taken until normal ACTH and cortisol production resumes.
Less commonly, adrenal insufficiency occurs when the pituitary gland either decreases in size or stops producing ACTH. These events can result from
tumors or infections of the area
loss of blood flow to the pituitary
radiation for the treatment of pituitary tumors
surgical removal of parts of the hypothalamus
surgical removal of the pituitary gland
How is adrenal insufficiency diagnosed?
In its early stages, adrenal insufficiency can be difficult to diagnose. A review of a patient’s medical history and symptoms may lead a doctor to suspect Addison’s disease.
A diagnosis of adrenal insufficiency is confirmed through laboratory tests. The aim of these tests is first to determine whether levels of cortisol are insufficient and then to establish the cause. Radiologic exams of the adrenal and pituitary glands also are useful in helping to establish the cause.
ACTH Stimulation Test
The ACTH stimulation test is the most commonly used test for diagnosing adrenal insufficiency. In this test, blood cortisol, urine cortisol, or both are measured before and after a synthetic form of ACTH is given by injection. The normal response after an ACTH injection is a rise in blood and urine cortisol levels. People with Addison's disease or long-standing secondary adrenal insufficiency have little or no increase in cortisol levels.
Both low- and high-dose ACTH stimulation tests may be used depending on the suspected cause of adrenal insufficiency. For example, if secondary adrenal insufficiency is mild or of recent onset, the adrenal glands may still respond to ACTH because they have not yet atrophied. Some studies suggest a low dose-1 microgram-may be more effective in detecting secondary adrenal insufficiency because the low dose is still enough to raise cortisol levels in healthy people but not in people with mild or recent secondary adrenal insufficiency.
CRH Stimulation Test
When the response to the ACTH test is abnormal, a CRH stimulation test can help determine the cause of adrenal insufficiency. In this test, synthetic CRH is injected intravenously and blood cortisol is measured before and 30, 60, 90, and 120 minutes after the injection. People with Addison’s disease respond by producing high levels of ACTH but no cortisol. People with secondary adrenal insufficiency have absent or delayed ACTH responses. CRH will not stimulate ACTH secretion if the pituitary is damaged, so an absent ACTH response points to the pituitary as the cause. A delayed ACTH response points to the hypothalamus as the cause.
Diagnosis during an Emergency
In patients suspected of having an Addisonian crisis, health professionals must begin treatment with injections of salt, glucose-containing fluids, and glucocorticoid hormones immediately. Although a reliable diagnosis is not possible during crisis treatment, measurement of blood ACTH and cortisol during the crisis-before glucocorticoids are given-is enough to make a preliminary diagnosis. Low blood sodium, low blood glucose, and high blood potassium are also usually present at the time of an adrenal crisis. Once the crisis is controlled, an ACTH stimulation test can be performed to obtain the specific diagnosis. More complex laboratory tests are sometimes used if the diagnosis remains unclear.
Other Tests
Once a diagnosis of Addison's disease is made, radiologic studies such as an x ray or an ultrasound of the abdomen may be taken to see if the adrenals have any signs of calcium deposits. Calcium deposits may indicate bleeding in the adrenal gland or TB, for which a tuberculin skin test also may be used. Blood tests can detect antibodies associated with autoimmune Addison's disease.
If secondary adrenal insufficiency is diagnosed, doctors may use different imaging tools to reveal the size and shape of the pituitary gland. The most common is the computerized tomography (CT) scan, which produces a series of x-ray pictures giving cross-sectional images. A magnetic resonance imaging (MRI) scan may also be used to produce a three-dimensional image of this region. The function of the pituitary and its ability to produce other hormones also are assessed with blood tests.
How is adrenal insufficiency treated?
Treatment of adrenal insufficiency involves replacing, or substituting, the hormones that the adrenal glands are not making. Cortisol is replaced with a synthetic glucocorticoid such as hydrocortisone, prednisone, or dexamethasone, taken orally once to three times each day, depending on which medication is chosen. If aldosterone is also deficient, it is replaced with oral doses of a mineralocorticoid, called fludrocortisone acetate (Florinef), taken once or twice a day. Doctors usually advise patients receiving aldosterone replacement therapy to increase their salt intake. Because people with secondary adrenal insufficiency normally maintain aldosterone production, they do not require aldosterone replacement therapy. The doses of each medication are adjusted to meet the needs of the individual.
During an Addisonian crisis, low blood pressure, low blood glucose, and high levels of potassium can be life threatening. Standard therapy involves intravenous injections of glucocorticoids and large volumes of intravenous saline solution with dextrose, a type of sugar. This treatment usually brings rapid improvement. When the patient can take fluids and medications by mouth, the amount of glucocorticoids is decreased until a maintenance dose is reached. If aldosterone is deficient, maintenance therapy also includes oral doses of fludrocortisone acetate.
What special problems can occur with adrenal insufficiency?
Surgery
Because cortisol is a "stress hormone," people with chronic adrenal insufficiency who need any type of surgery requiring general anesthesia must be treated with intravenous glucocorticoids and saline. Intravenous treatment begins before surgery and continues until the patient is fully awake after surgery and able to take medication by mouth. The "stress" dosage is adjusted as the patient recovers until the presurgery maintenance dose is reached.
In addition, people who are not currently taking glucocorticoids but who have taken long-term glucocorticoids in the past year should tell their doctor before surgery. These people may have sufficient ACTH for normal events, but they may need intravenous treatment for the stress of surgery.
Illness
During illness, oral dosing of glucocorticoid may be adjusted to mimic the normal response of the adrenal glands to this stress on the body. Significant fever or injury may require triple oral dosing. Once recovery from the stress event is achieved, dosing is then returned to maintenance levels. People with adrenal insufficiency should know how to increase medication during such periods of stress. Immediate medical attention is needed if severe infections, vomiting, or diarrhea occur. These conditions can precipitate an Addisonian crisis.
Pregnancy
Women with adrenal insufficiency who become pregnant are treated with standard replacement therapy. If nausea and vomiting in early pregnancy interfere with taking medication by mouth, injections of the hormone may be necessary. During delivery, treatment is similar to that of people needing surgery. Following delivery, the dose is gradually tapered and the usual maintenance doses of oral hydrocortisone and fludrocortisone acetate are reached about 10 days after childbirth.
How can someone with adrenal insufficiency prepare for an emergency?
People with adrenal insufficiency should always carry identification stating their condition in case of an emergency. A card or medical alert tag should notify emergency health care providers of the need to inject cortisol if the person is found severely injured or unable to answer questions. The card or tag should also include the name and telephone number of the person’s doctor and the name and telephone number of a family member to be notified. The dose of hydrocortisone needed may vary with a person's age or size. For example, a child younger than 2 years of age can receive 25 milligrams (mg), a child between 2 and 8 years of age can receive 50 mg, and a child older than 8 years should receive the adult dose of 100 mg. When traveling, people with adrenal insufficiency should carry a needle, syringe, and an injectable form of cortisol for emergencies.
Points to Remember
Adrenal insufficiency is a disorder that occurs when the adrenal glands do not produce enough of certain hormones.
Primary adrenal insufficiency, also called Addison's disease, occurs when the adrenal glands are damaged and cannot produce enough of the hormone cortisol and often the hormone aldosterone.
Secondary adrenal insufficiency occurs when the pituitary gland fails to produce enough adrenocorticotropin (ACTH), a hormone that stimulates the adrenals to produce cortisol. If ACTH output is too low, cortisol production drops.
The most common symptoms of adrenal insufficiency are chronic, worsening fatigue; muscle weakness; loss of appetite; and weight loss.
Adrenal insufficiency is most often diagnosed through blood or urine tests. Imaging studies such as x rays, ultrasound, computerized tomography (CT), and magnetic resonance imaging (MRI) may also be used.
Treatment of adrenal insufficiency involves replacing, or substituting, the hormones that the adrenal glands are not making.
People with adrenal insufficiency should always carry identification stating their condition in case of an emergency.
Hope through Research
Researchers are evaluating the use of another adrenal hormone, dehydroepiandrosterone, in improving the health-related quality of life in people with adrenal insufficiency. Scientists are also studying the occurrence of "relative" adrenal insufficiency-below normal production of adrenal hormones during critical illness-to further define this type of adrenal insufficiency, develop diagnostic tests, and identify the best treatment options.
Participants in clinical trials can play a more active role in their own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research. For information about current studies, visit www.ClinicalTrials.gov.
For More Information
The following articles about Addison's disease can be found in medical libraries, some college and university libraries, and through interlibrary loan in most public libraries.
Chrousos GP. Glucocorticoid therapy. In: Felig P, Frohman L, eds. Endocrinology and Metabolism. 4th ed. New York: McGraw-Hill; 2001:609–632.
Munver R, Volfson IA. Adrenal insufficiency: diagnosis and management. Current Urology Reports. 2006;7:80-85.
Nieman LK, Chanco Turner ML. Addison's disease. Clinics in Dermatology. 2006;24(4):276-280.
Stewart PM. The adrenal cortex. In: Larsen P, ed. Williams Textbook of Endocrinology. 10th ed. Philadelphia: Saunders; 2003:491-551.
Ten S, New M, Maclaren N. Clinical Review 130: Addison's disease 2001. Journal of Clinical Endocrinology & Metabolism. 2001;86(7):2909-2922.
Williams GH, Dluhy RC. Disorders of the adrenal cortex. In: Braunwald E, ed. Harrison's Principles of Internal Medicine. 17th ed. New York: McGraw-Hill Professional; 2008:2247-2268.
The following organizations might also be able to assist with certain types of information:
American Autoimmune Related Diseases Association
National Office
22100 Gratiot Avenue
East Detroit, MI 48021
Phone: 586-776-3900
Email: aarda@aol.com
Internet: www.aarda.org
National Adrenal Diseases Foundation
505 Northern Boulevard, Suite 200
Great Neck, NY 11021
Phone: 516-487-4992
Email: NADFmail@aol.com
Internet: www.nadf.us
You may also find additional information about this topic by visiting MedlinePlus at www.medlineplus.gov.
Acknowledgments
Publications produced by the NIDDK are carefully reviewed by both NIDDK scientists and outside experts. This publication was reviewed by Karen Loechner, M.D., Ph.D., University of North Carolina at Chapel Hill.
National Endocrine and Metabolic Diseases Information Service
6 Information Way
Bethesda, MD 20892–3569
Phone: 1–888–828–0904
TTY: 1–866–569–1162
Fax: 703–738–4929
Email: endoandmeta@info.niddk.nih.gov
Internet: www.endocrine.niddk.nih.gov
The National Endocrine and Metabolic Diseases Information Service is an information dissemination service of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). The NIDDK is part of the National Institutes of Health, which is part of the U.S. Department of Health and Human Services.
The NIDDK conducts and supports biomedical research. As a public service, the NIDDK has established information services to increase knowledge and understanding about health and disease among patients, health professionals and the public.
May 2009
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Adrenal Gland Disorders
What are the adrenal glands?
The adrenal glands are the part of the body responsible for releasing three different classes of hormones. These hormones control many important functions in the body, such as:
Maintaining metabolic processes, such as managing blood sugar levels and regulating inflammation
Regulating the balance of salt and water
Controlling the "fight or flight" response to stress
Maintaining pregnancy
Initiating and controlling sexual maturation during childhood and puberty
The adrenal glands are also an important source of sex steroids, such as estrogen and testosterone.
What are adrenal gland disorders?
Adrenal gland disorders occur when the adrenal glands don’t work properly. Sometimes, the cause is a problem in another gland that helps to regulate the adrenal gland. In other cases, the adrenal gland itself may have the problem. The NICHD conducts and supports research on many adrenal gland disorders. Some examples include:
Cushing's Syndrome - Cushing’s syndrome happens when a person’s body is exposed to too much of the hormone cortisol. In this syndrome, a person’s body makes more cortisol than it needs. For example, adrenal tumors can cause the body to produce too much cortisol. In some cases, children are born with a form of adrenal hyperplasia that leads to Cushing syndrome. Or, in some cases, certain medications can cause the body to make too much cortisol
Congenital Adrenal Hyperplasia - Congenital adrenal hyperplasia is a genetic disorder of adrenal gland deficiency. In this disorder, the body doesn’t make enough of the hormone cortisol. The bodies of people with congenital adrenal hyperplasia may also have other hormone imbalances, such as not making enough aldosterone, but making too much androgen.
Pituitary Tumors - The pituitary gland is located in the brain and helps to regulate the activity of most other glands in the body, including the adrenal glands. In rare cases, benign (non-cancerous) tumors may grow on the pituitary gland, which may restrict the hormones it releases.
In some cases, tumors on the pituitary can lead to Cushing’s syndrome – this is called Cushing disease. In other cases, the tumors reduce the adrenal gland’s release of hormones needed for the “fight or flight” response to stress. If the body is unable to handle physiological stress—a condition called Addison’s disease—it can be fatal.
What are the treatments for adrenal gland disorders?
The treatment for adrenal gland disorders depends on the specific disorder or the specific cause of the disorder. For example:
The treatment for Cushing’s syndrome depends on the cause. If the excess cortisol is caused by medication, your health care provider can change dosages or try a different medication to correct the problem. If the Cushing’s syndrome is caused by the body making too much cortisol, treatments may include oral medication, surgery, radiation, or a combination of these treatments.
Congenital adrenal hyperplasia can’t be cured, but it can be treated and controlled. People with congenital adrenal hyperplasia can take medication to help replace the hormones their bodies are not making. Some people with congenital adrenal hyperplasia only need these medications when they are sick, but others may need to take them every day.
Doctors can successfully treat most pituitary tumors with microsurgery, radiation therapy, surgery, drugs, or a combination of these treatments. Surgery is currently the treatment of choice for tumors that grow rapidly, especially if they threaten or affect vision. The treatment plan for other pituitary tumors differs according to the type and size of the tumor.
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NINDS Orthostatic Hypotension Information
What is Orthostatic Hypotension?
Orthostatic hypotension is a sudden fall in blood pressure that occurs when a person assumes a standing position. It may be caused by hypovolemia (a decreased amount of blood in the body), resulting from the excessive use of diuretics, vasodilators, or other types of drugs, dehydration, or prolonged bed rest. The disorder may be associated with Addison's disease, atherosclerosis (build-up of fatty deposits in the arteries), diabetes, and certain neurological disorders including Shy-Drager syndrome and other dysautonomias. Symptoms, which generally occur after sudden standing, include dizziness, lightheadedness, blurred vision, and syncope (temporary loss of consciousness).
Is there any treatment?
When orthostatic hypotension is caused by hypovolemia due to medications, the disorder may be reversed by adjusting the dosage or by discontinuing the medication. When the condition is caused by prolonged bed rest, improvement may occur by sitting up with increasing frequency each day. In some cases, physical counterpressure such as elastic hose or whole-body inflatable suits may be required. Dehydration is treated with salt and fluids.
What is the prognosis?
The prognosis for individuals with orthostatic hypotension depends on the underlying cause of the condition.
What research is being done?
The NINDS supports research on conditions such as neurogenic orthostatic hypotension aimed at increasing scientific understanding of the condition and finding ways to treat and prevent it.
Organizations
National Organization for Rare Disorders (NORD)
P.O. Box 1968
(55 Kenosia Avenue)
Danbury, CT 06813-1968
orphan@rarediseases.org
www.rarediseases.org
Tel: 203-744-0100 Voice Mail 800-999-NORD (6673)
Fax: 203-798-2291
National Heart, Lung, and Blood Institute (NHBLI)
National Institutes of Health, DHHS
31 Center Drive, Rm. 4A21 MSC 2480
Bethesda, MD 20892-2480
www.nhlbi.nih.gov
Tel: 301-592-8573/240-629-3255 (TTY) Recorded Info: 800-575-WELL (-9355)
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Directory of Endocrine and Metabolic Diseases Organizations
This directory lists Government agencies and professional and voluntary associations that provide endocrine and metabolic diseases-related information and resources. Some of these organizations offer educational materials and other services to patients and the general public, while others primarily serve health care professionals
American Academy of Otolaryngology-Head and Neck Surgery (AAO-HNS)
1650 Diagonal Road
Alexandria, VA 22314-2857
Phone: 703-836-4444
Internet: www.entnet.org
Mission: To advance the art, science, and ethical practice of otolaryngology-head and neck surgery through education, research, and lifelong learning.
Materials: Otolaryngology-Head and Neck Surgery (journal); Bulletin (monthly magazine); physician educational materials and courses; extensive patient education materials; website search capability for locating head and neck surgeons and physicians.
American Association of Clinical Endocrinologists (AACE)
245 Riverside Avenue, Suite 200
Jacksonville, FL 32202
Phone: 904-353-7878
Fax: 904-353-8185
Internet: www.aace.com
Mission: To provide a professional community of clinical endocrinologists that is committed to transforming the lives of patients by enabling one another to practice leading-edge, proactive, ethical, and cost-effective medicine.
Materials: Endocrine Practice (journal); The First Messenger (newsletter); practice guidelines.
The American Porphyria Foundation (APF)
4900 Woodway, Suite 780
Houston, TX 77056-1837
Phone: 1-866-APF-3635 (1-866-273-3635) or 713-266-9617
Fax: 713-840-9552
Email: porphyrus@aol.com
Internet: www.porphyriafoundation.com
Mission: To enhance public awareness about porphyria, develop and distribute educational materials for patients and physicians, and support research to improve treatment and lead to a cure. The APF also serves as an advocate to other public, private, and government agencies interested in funding research and educational programs.
Materials: Acute Intermittent Porphyria, Porphyria Cutanea Tarda (PCT), ALAD Porphyria (ADP), Hepatoerythropoietic Porphyria (HEP), Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), and Erythropoietic Protoporphyria (EPP) (informational brochures); newsletter.
American Society for Bone and Mineral Research (ASBMR)
2025 M Street NW, Suite 800
Washington, DC 20036
Phone: 202-367-1161
Fax: 202-367-2161
Email: asbmr@asbmr.org
Internet: www.asbmr.org
Mission: To promote excellence in bone and mineral research, foster integration of clinical and basic science, and facilitate the translation of that science to health care and clinical practice.
Materials: Journal of Bone and Mineral Research (monthly journal); Primer on the Metabolic Bone Diseases and Disorders of Mineral Metabolism (6th edition); ASBMR e-news (monthly e-newsletter).
American Society of Human Genetics (ASHG)
9650 Rockville Pike
Bethesda, MD 20814
Phone: 1-866-HUM-GENE (1-866-486-4363) or 301-571-1825
Email: society@ashg.org
Internet: www.ashg.org
Mission: To promote and expand research, apply knowledge to enhance health care, train the next generation of professionals, and educate and inform the public about genetics.
Materials: American Journal of Human Genetics (monthly journal); Enhancement of K–12 Human Genetics Education: Creating a Cooperative Plan; Guide to Human Genetics Training Programs.
American Thyroid Association (ATA)
6066 Leesburg Pike, Suite 550
Falls Church, VA 22041
Phone: 1-800-THYROID (1-800-849-7643) or 703-998-8890
Fax: 703-998-8893
Email: thyroid@thyroid.org
Internet: www.thyroid.org
Mission: To provide outstanding leadership in thyroidology by promoting excellence and innovation in clinical care, research, education, and public policy.
Materials: Informational pamphlets; Thyroid (monthly journal); Clinical Thyroidology (summaries of published thyroid studies, published three times a year); The Signal (newsletter for members, published three times a year).
Association for Glycogen Storage Disease (AGSD)
P.O. Box 896
Durant, IA 52747
Phone: 563-785-6038
Email: maryc@agsdus.org
Internet: www.agsdus.org
Mission: To serve as a parent- and patient-oriented support group, increase public awareness of glycogen storage disease (GSD), stimulate research in the various forms of GSD, and disseminate results to parents, patients, and the public.
Materials: The Ray (periodic newsletter); Parent Handbook; other brochures.
Congenital Adrenal Hyperplasia Research Education and Support (CARES) Foundation
2414 Morris Avenue
Union, NJ 07083
Phone: 1-866-227-3737 or 973-912-3895
Internet: www.caresfoundation.org
Mission: To improve the lives of families and patients affected by congenital adrenal hyperplasia (CAH) by proactively advancing research for a cure, educating the public and health care professionals about all forms of CAH, advocating for universal newborn screening, and providing support services and resources vital to the CAH community worldwide.
Materials: Newsletters; brochures; newborn screening packets.
Cushing's Support and Research Foundation (CSRF)
65 East India Row, 22B
Boston, MA 02110
Phone: 617-723-3674
Email: cushinfo@csrf.net
Internet: www.csrf.net
Mission: To provide information and support for patients and their families, increase awareness and educate the public about Cushing's disease and Cushing's syndrome, be a resource for information and support to health care professionals, and raise and distribute funds for Cushing's disease and Cushing's syndrome research.
Materials: Quarterly newsletter; networking contacts list; CSRF Special Newsletter with personal stories; brochures; fact sheets; listings of Cushing's specialists by state.
Cystic Fibrosis Foundation
6931 Arlington Road
Bethesda, MD 20814
Phone: 1-800-FIGHT-CF (1-800-344-4823) or 301-951-4422
Fax: 301-951-6378
Email: info@cff.org
Internet: www.cff.org
Mission: To develop more therapies that will extend and enhance the lives of those with cystic fibrosis (CF) and to find a cure or control for CF.
Materials: Informational brochures; videos.
Endocrine Society
8401 Connecticut Avenue, Suite 900
Chevy Chase, MD 20815-5817
Phone: 1-888-363-274 or 301-941-0200
Fax: 301-941-0259
Email: societyservices@endo-society.org
Internet: www.endo-society.org
Mission: To promote excellence in research, education, and the clinical practice of endocrinology; to foster a greater understanding of endocrinology among the general public and practitioners of complementary medical disciplines; and to promote the interests of all endocrinologists at the national scientific research and health policy levels of Government.
Materials: Endocrine Reviews, Endocrinology, The Journal of Clinical Endocrinology and Metabolism, and Molecular Endocrinology (journals); Molecular and Cellular Research Syllabi, Program and Abstracts Book, Recent Progress in Hormone Research Conference Proceedings, Clinical Endocrinology Update Syllabi, Remembrances in Endocrinology, and Endocrine Reviews Monograph Series (books); Endocrine News (monthly magazine).
Fatty Oxidation Disorders (FOD) Family Support Group
Deb Lee Gould, M.Ed.
2041 Tomahawk
Okemos, MI 48864
Phone: 517-381-1940
Fax(United States/Canada) 1-866-290-5206
Fax(Overseas) 313-432-5928
Email: deb@fodsupport.org
Internet: www.fodsupport.org
Mission: To connect and network with fatty oxidation disorders (FOD) families and professionals around the world and to provide emotional support, family stories, practical information about living with these disorders, and medical updates to inform families of new developments in screening, diagnosis, research, and treatment.
Materials: FOD Communication Network (biannual newsletter) for families and professionals.
The Genetic Alliance
4301 Connecticut Avenue, NW Suite 404
Washington, DC 20008-2369
Phone: 1-800-336-GENE (4363) or 202-966-5557
Fax: 202-966-8553
Email: info@geneticalliance.org
Internet: www.geneticalliance.org
Mission: To increase the capacity of genetic advocacy organizations to achieve their missions and leverage the voices of millions of individuals and families living with genetic conditions.
Materials: Newsletter; general brochure; Bibliography of Material on Basic Genetics and DNA Technology for the General Public, Directory of National Genetic Voluntary Organizations, Integrating Consumers into Regional Genetics Networks, Informed Consent—Participation in Genetic Research Studies, Media Reporting in the Genetic Age, and Starting a Support Group (brochures).
Graves' Disease Foundation (GDF)
400 International Drive
Williamsville, NY 14221
Phone: 1-877-643-3123 or 716-631-2310
Fax: 716-631-2822
Email: gravesdiseasefd@gmail.com
Internet: www.ngdf.org
Mission: To provide medical information, referrals, and resources to patients; aid in the development of support groups; provide professional education through lectures and forums; and foster research on Graves’ disease.
Materials: Informational brochures; periodic bulletins.
The Hormone Foundation
8401 Connecticut Avenue, Suite 900
Chevy Chase, MD 20815-5817
Phone: 1-800-HORMONE (1-800-467-6663)
Fax: 301-941-0259
Email: hormone@endo-society.org
Internet: www.hormone.org
Mission: To serve as a resource for the public by promoting the prevention, treatment, and cure of hormone-related conditions through outreach and education. The Hormone Foundation is the public education affiliate of the Endocrine Society.
Materials: Patient fact sheets; brochures; Recognition and Treatment of Pituitary Hormone Imbalances (video/webcast).
Human Growth Foundation (HGF)
997 Glen Cove Avenue, Suite 5
Glen Head, NY 11545
Phone: 1-800-451-6434
Fax: 516-671-4055
Email: hgf1@hgfound.org
Internet: www.hgfound.org
Mission: To support families of children with physical growth problems and to help medical science better understand the process of growth. The HGF distributes funds for basic and clinical growth research.
Materials: Fourth Friday (quarterly newsletter); Growth Series (brochures).
Hypoglycemia Support Foundation, Inc. (HSF)
P.O. Box 451778
Sunrise, FL 33345
Phone: 954-742-3098
Email: rruggiero@hypoglycemia.org
Internet: www.hypoglycemia.org
Mission: To inform, direct, support, and encourage people with hypoglycemia.
Materials: The Blood Sugar Hotline (CD); Hypoglycemia: Millennium Update (audiotapes); The Health Emergency Card; The Do's and Don'ts of Hypoglycemia (book); Seven Day Diet Menu.
Iron Overload Diseases Association (IOD)
P.O. Box 15857
West Palm Beach, FL 33416
Phone: 1-866-768-8629 or 561-586-8246
Fax: 561-586-8248
Email: iod@ironoverload.org
Internet: www.ironoverload.org
Mission: To conduct professional education symposia and exhibits at medical meetings; serve and counsel patients with hemochromatosis and their families; offer doctor referrals; promote patient advocacy concerning insurance, Medicare, blood banks, and the U.S. Food and Drug Administration; encourage research; maintain an international clearinghouse; offer public information through the media; develop chapters and self-help groups; and sponsor annual symposia and IOD Awareness Week.
Materials: Overload: An Ironic Disease (booklet); Ironic Blood (bimonthly newsletter); Iron Overload Alert (informational brochure); fact sheets.
The Magic Foundation
6645 West North Avenue
Oak Park, IL 60302
Phone: 1-800-3-MAGIC3 (1-800-362-4423) or 708-383-0808
Fax: 708-383-0899
Email: dianne@magicfoundation.org
Internet: www.magicfoundation.org
Mission: To provide support services for the families of children afflicted with a wide variety of chronic and/or critical disorders, syndromes, and diseases that affect a child's growth.
Materials: Educational brochures; The MAGIC Touch (quarterly newsletter); The MAGIC Star (quarterly newsletter for adults with growth hormone deficiency); newsletter for affected children; National Pen Pal program.
March of Dimes
1275 Mamaroneck Avenue
White Plains, NY 10605
Phone: 1-888-MODIMES (1-888-663-4637) or 914-997-4488
Fax: 914-428-8203
Email: askus@marchofdimes.com
Internet: www.marchofdimes.com
Mission: To improve the health of babies by preventing birth defects, premature birth, and infant mortality through research, community services, education, and advocacy.
Materials: Educational materials about prenatal care, birth defects, and prematurity; nursing modules; monthly newsletter; newborn screening recommendations.
National Adrenal Diseases Foundation (NADF)
505 Northern Boulevard
Great Neck, NY 11021
Phone: 516-487-4992
Email: NADFmail@aol.com
Internet: www.nadf.us
Mission: To provide support, information, and education to individuals with Addison’s disease and other diseases of the adrenal glands.
Materials: NADF Newsletter (quarterly newsletter); educational materials.
National Diabetes Information Clearinghouse (NDIC)
1 Information Way
Bethesda, MD 20892–3560
Phone: 1-800-860-8747
TTY: 1-866-569-1162
Fax: 703-738-4929
Email: ndic@info.niddk.nih.gov
Internet: www.diabetes.niddk.nih.gov
Mission: To serve as a diabetes information, education, and referral resource for health professionals and the public. The NDIC is a service of the NIDDK.
Materials: Educational materials about diabetes, available free or at little cost; literature searches on a variety of subjects related to diabetes; Diabetes Dateline (e-newsletter).
National MPS Society
P.O. Box 14686
Durham, NC 27709-4686
Phone: 919-8067-0101
Fax: 919-806-2055
Email: info@mpssociety.org
Internet: www.mpssociety.org
Mission: To support families of children with mucopolysaccharidoses (MPS) and related diseases, increase professional and public awareness of the disorders, and raise funds to further research on MPS and related disorders.
Materials: Courage (quarterly newsletter); informational booklets.
National Organization for Rare Disorders (NORD)
55 Kenosia Avenue, P.O. Box 1968
Danbury, CT 06813-1968
Phone: 1-800-999-6673 or 203-744-0100
TDD: 203-797-9590
Fax: 203-798-2291
Email: orphan@rarediseases.org or RN@rarediseases.org
Internet: www.rarediseases.org
Mission: To help people with rare "orphan" diseases and assist the organizations that serve them. The NORD is committed to the identification, treatment, and cure of rare disorders through education, advocacy, research, and service programs.
Materials: Orphan Disease Update (member newsletter); NORD Resource Guide; Physicians' Guide to Rare Diseases; free booklets for physicians about several rare disorders.
National Osteoporosis Foundation (NOF)
1232 22nd Street NW
Washington, DC 20037
Phone: 1-800-231-4222 or 202-223-2226
Internet: www.nof.org
Mission: To prevent osteoporosis; promote lifelong bone health; help improve the lives of those affected by osteoporosis and related fractures; and find a cure through programs of awareness, education and training, advocacy, and research.
Materials: Osteoporosis Report (quarterly newsletter); informational brochures; books; videos; other educational materials.
National Urea Cycle Disorders Foundation (NUCDF)
4841 Hill Street
La Canada, CA 91011
Phone: 1-800-38-NUCDF (1-800-386-8233)
Fax: 818-952-2184
Email: info@nucdf.org
Internet: www.nucdf.org
Mission: To provide a primary resource of information and education to families and medical professionals and to support and stimulate research on urea cycle disorders.
Materials: Informational brochures; quarterly newsletter.
Organic Acidemia Association, Inc. (OAA)
13210 35th Avenue North
Plymouth, MN 55441
Phone: 763-559-1797
Fax: 1-866-539-4060
Email: OAANews@aol.com
Internet: www.oaanews.org
Mission: To educate families and health care professionals about organic acidemia metabolic disorders, support early intervention through expanded newborn screening, and solicit contributions and distribute funding that supports research toward improved treatment and eventual cures for organic acid disorders.
Materials: Organic Acidemia Association Newsletter (quarterly newsletter).
Oxalosis and Hyperoxaluria Foundation (OHF)
201 East 19th Street, Suite 12E
New York, NY 10003
Phone: 1-800-OHF-8699 (1-800-643-8699) or 212-777-0470
Fax: 212-777-0471
Email: kimh@ohf.org
Internet: www.ohf.org
Mission: To promote research to find a cure for oxalosis, primary hyperoxaluria, and related stone diseases and improve the care and treatment of those they affect.
Materials: Patient Handbook; In Touch (newsletter); website resources.
The Paget Foundation for Paget's Disease of Bone and Related Disorders
120 Wall Street, Suite 1602
New York, NY 10005-4001
Phone: 1-800-23-PAGET (1-800-237-2438) or 212-509-5335
Fax: 212-509-8492
Email: PagetFdn@aol.com
Internet: www.paget.org
Mission: To serve patients with Paget's disease of bone, primary hyperparathyroidism, and other related disorders. The Paget Foundation also assists the medical community that treats patients.
Materials: Quarterly newsletter; patient education brochures.
The Pituitary Network Association (PNA)
P.O. Box 1958
Thousand Oaks, CA 91358
Phone: 805-499-9973
Fax: 805-480-0633
Email: pna@pituitary.org
Internet: www.pituitary.org
Mission: To support, pursue, encourage, promote, and, where possible, fund research on pituitary disorders in a sustained and full-time effort to find a cure for these illnesses. The PNA disseminates information about early detection, symptoms, treatments, and available resources to the medical community, the public, and pituitary patients and their families.
Materials: Network (quarterly newsletter); informational pamphlets; The Pituitary Patient Resource Guide.
The Pituitary Society
VA Medical Center
423 East 23rd Street, Room 16048aW
New York, NY 10010
Phone: 212-263-6772
Fax: 212-447-6219
Internet: www.pituitarysociety.org
Mission: To improve the health of individuals with pituitary disorders through scientific and educational programs for patients, their families, medical students, scientists, and physicians. The Pituitary Society also encourages the development of research programs to study the biology of the pituitary and the etiology, diagnosis, pathophysiology, and treatment of pituitary disease.
Materials: Pituitary (journal); online patient education materials about the pituitary gland and specific disorders.
Society for Inherited Metabolic Disorders (SIMD)
Leslie Lublink, SIMD Administrator
17007 Old River Drive
Lake Oswego, OR 97034
Phone: 503-636-9228
Fax: 503-210-1511
Email: lublinkl@ohsu.edu
Internet: www.simd.org
Mission: To advise the Government on the use of medical products for the treatment of inborn errors of metabolism, establish a connected group of centers for the diagnosis and treatment of patients with metabolic diseases, foster research, and ensure the availability of expertise for diagnosis and treatment of patients with inherited metabolic disorders.
Materials: Molecular Genetics & Metabolism (journal).
United Leukodystrophy Foundation (ULF)
2304 Highland Drive
Sycamore, IL 60178
Phone: 1-800-728-5483
Fax: 815-895-2432
Email: office@ulf.org
Internet: www.ulf.org
Mission: To help children and adults with leukodystrophy and assist the family members, professionals, and support services that serve them. The ULF also supports the identification, treatment, and cure of all leukodystrophies through programs of education, advocacy, research, and service.
Materials: Fact sheets about individual leukodystrophies; ULF News (quarterly newsletter).
Wilson's Disease Association International (WDA)
1802 Brookside Drive
Wooster, OH 44691
Phone: 1-888-264-1450 or 330-264-1450
Fax: 330-264-0974
Email: info@wilsonsdisease.org
Internet: www.wilsonsdisease.org
Mission: To fund research and facilitate and promote the identification, education, treatment, and support of patients and other individuals affected by Wilson disease.
Materials: The Copper Connection (newsletter); informational brochures.
National Endocrine and Metabolic Diseases Information Service
6 Information Way
Bethesda, MD 20892-3569
Phone: 1-888-828-0904
TTY: 1-866-569-1162
Fax: 1-703-738-4929
Email: endoandmeta@info.niddk.nih.gov
Internet: endocrine.niddk.nih.gov/
The National Endocrine and Metabolic Diseases Information Service is an information dissemination service of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). The NIDDK is part of the National Institutes of Health, which is part of the U.S. Department of Health and Human Services.
* * * * * * * * * * * *
ADDISON'S DISEASE CLINICAL TRIALS
www.clinicaltrials.gov - Retrieved July 2011
Rank * Status († indicates that status has not been verified in more than two years) * Study
* * * * * * * * * * * *
1 * Completed * Effects and Interactions of Liquorice and Grapefruit on Glucocorticoid Replacement Therapy in Addison's Disease
Condition: Addison Disease
Interventions: Dietary Supplement: Liquorice; Dietary Supplement: Grapefruit Juice
Sponsor: Haukeland University Hospital
Gender: Both
Age Groups: Adult / Senior
Phase:
Number Enrolled: 17
Funded By: Other
Study Type: Interventional
Study Design: Allocation: Randomized; Endpoint Classification: Pharmacokinetics Study; Intervention Model: Crossover Assignment; Masking: Open Label; Primary Purpose: Supportive Care
NCT ID: NCT01271296
Other IDs: 3.2007.2620 (REK), 17775 (NSD), 07/5829 (SHdir), 3.2007.2620
First Received Date: December 22, 2010
Start Date: April 2008
Completion Date: January 2009
Last Updated Date: January 13, 2011
Last Verified Date: December 2010
Acronym:
Primary Completion Date: January 2009
Outcome Measures: AUC Serum Cortisol - Levels of cortisol in serum during the first 2.6 hours after oral administration of cortisone acetate.; Serum Cortisol levels at the end of time-series sampling (t=160min); Serum Cortisone levels at the end of time-series sampling (t=160min); Saliva Cortisol levels at the end of time-series sampling (t=160min); Saliva Cortisone levels at the end of time-series sampling (t=160min); Time of maximum concentration of serum Cortisol; Time of maximum concentration of serum Cortisone; Time of maximum concentration of Saliva Cortisol; Time of maximum concentration of Saliva Cortisone; Half life of serum cortisol; Half life of serum cortisone; Urinary aTHF/THF-ratio; AUC Serum Cortisone; Urine total metabolites (Urinary cortisol+cortisone+6OHF+aTHF+THF+THE); Urinary ratio (aTHF+THF)/THE; Urinary Ratio Cortisol/6beta-OH-Cortisol; AUC Saliva cortisone; AUC Saliva Cortisol
* * * * * * * * * * * *
2 * Enrolling by invitation * Glucocorticoid Treatment in Addison's Disease
Condition: Addison's Disease
Interventions: Drug: Solu-Cortef (hydrocortisone); Drug: Cortef (hydrocortisone)
Sponsor: Haukeland University Hospital
Gender: Both
Age Groups: Adult / Senior
Phases: Phase II / Phase III
Number Enrolled: 40
Funded By: Other
Study Type: Interventional
Study Design: Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Crossover Assignment; Masking: Open Label; Primary Purpose: Treatment
NCT ID: NCT01063569
Other IDs: 3.2007.2343, 2009-010917-61
First Received Date: January 26, 2010
Start Date: February 2010
Completion Date: October 2012
Last Updated Date: September 12, 2010
Last Verified Date: September 2010
Acronym:
Primary Completion Date: October 2012
Outcome Measures: Morning (08-09) plasma ACTH; Health-Related Quality of Life; evaluated by SF-36 vitality scores and AddiQoL scores; Sleep; evaluated by Pittsburgh Sleep Quality Index (PSQI), and 7-days' Actigraph registration combined with self-reported sleep diary; 24h cortisol profiles in serum and saliva; S-glucose, 24 h profile; Insulin sensitivity assessed by euglycemic clamp
3 * Completed * Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dehydroepiandrosterone Replacement for Primary Adrenal Insufficiency
Condition: Addison's Disease
Intervention: Drug: dehydroepiandrosterone
Sponsors: National Center for Research Resources (NCRR); University of California, Los Angeles
Gender: Both
Age Groups: Adult / Senior
Phase: Phase III
Number Enrolled: 40
Funded By: NIH / Other
Study Type: Interventional
Study Design: Allocation: Randomized; Endpoint Classification: Efficacy Study; Masking: Double-Blind; Primary Purpose: Treatment
NCT ID: NCT00004313
Other IDs: 199/11822, UCSD-1062
First Received Date: October 18, 1999
Start Date: August 1995
Completion Date:
Last Updated Date: June 23, 2005
Last Verified Date: January 2000
Acronym:
Primary Completion Date:
Outcome Measure:
* * * * * * * * * * * *
4 * Completed * The Hypothalamic-Pituitary-Adrenal (HPA) Axis as an Effector System in Weight Regulation
Conditions: Obesity; Addison's Disease
Interventions: Drug: Hydrocortisone; Dietary Supplement: Isocaloric Diet
Sponsor: Oregon Health and Science University
Gender: Both
Age Groups: Adult / Senior
Phase:
Number Enrolled: 24
Funded By: Other
Study Type: Interventional
Study Design: Allocation: Randomized; Endpoint Classification: Pharmacokinetics/Dynamics Study; Intervention Model: Parallel Assignment; Masking: Single Blind (Investigator); Primary Purpose: Treatment
NCT ID: NCT00688987
Other IDs: eIRB 545, OCTRI #711
First Received Date: May 30, 2008
Start Date: August 2000
Completion Date: August 2004
Last Updated Date: June 4, 2008
Last Verified Date: June 2008
Acronym:
Primary Completion Date: July 2004
Outcome Measures: Amounts of intra-abdominal fat and total fat at the end of the treatment period for each cortisol dose.; Fasting Lipid levels, fat mass by DEXA, post-heparin lipase activity, insulin sensitivity, and fat biopsy
* * * * * * * * * * * *
5 * Completed * Survey in Patients With Chronic Adrenal Insufficiency in Germany
Conditions: Adrenal Insufficiency; Addison's Disease
Intervention:
Sponsor: University of Wuerzburg
Gender: Both
Age Groups: Child / Adult / Senior
Phase:
Number Enrolled:
Funded By: Other
Study Type: Observational
Study Design: Additional Descriptors: Psychosocial; Observational Model: Defined Population; Time Perspective: Cross-Sectional; Time Perspective: Retrospective/Prospective
NCT ID: NCT00444119
Other IDs: Wü NNRIS 45/04
First Received Date: March 5, 2007
Start Date: May 2004
Completion Date: April 2005
Last Updated Date: March 5, 2007
Last Verified Date: March 2007
Acronym:
Primary Completion Date:
Outcome Measure:
* * * * * * * * * * * *
6 * Unknown † * Adrenal Function After Living Kidney Donation
Conditions: Addison Disease; Hypoaldosteronism
Intervention: Procedure: Laparoscopic hand-assisted donor nephrectomy
Sponsors: University of Zurich; Ludwig-Maximilians - University of Munich
Gender: Both
Age Groups: Adult / Senior
Phase:
Number Enrolled: 30
Funded By: Other
Study Type: Observational
Study Design: Observational Model: Defined Population; Observational Model: Natural History; Time Perspective: Cross-Sectional; Time Perspective: Prospective
NCT ID: NCT00251836
Other IDs: StV 34-2005, RAB-F-02-0046
First Received Date: November 8, 2005
Start Date: December 2005
Completion Date: December 2007
Last Updated Date: April 17, 2007
Last Verified Date: April 2007
Acronym:
Primary Completion Date:
Outcome Measure:
* * * * * * * * * * * *
7 * Recruiting * Revival of Stem Cells in Addison's Study
Condition: Adrenal Failure
Intervention: Drug: depot tetracosactide
Sponsor: Newcastle University
Gender: Both
Age Groups: Child / Adult / Senior
Phase: Phase IV
Number Enrolled: 12
Funded By: Other
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
NCT ID: NCT01371526
Other IDs: RoSAv1.2:04 10: R&D#5252, 2009-018074-56
First Received Date: March 9, 2011
Start Date: September 2010
Completion Date: July 2012
Last Updated Date: June 9, 2011
Last Verified Date: June 2011
Acronym: RoSA
Primary Completion Date: July 2012
Outcome Measures: Peak serum Cortisol following ACTH stimulation; Change in QoL
* * * * * * * * * * * *
8 * Completed * Once-Daily Oral Modified-Release Hydrocortisone in Patients With Adrenal Insufficiency
Condition: Adrenal Insufficiency
Interventions: Drug: hydrocortisone (modified release), oral tablet 20 and 5 mg; Drug: Hydrocortisone, oral tablet, 10 mg
Sponsor: DuoCort AB
Gender: Both
Age Groups: Adult / Senior
Phases: Phase II / Phase III
Number Enrolled: 64
Funded By: Industry
Study Type: Interventional
Study Design: Allocation: Randomized; Endpoint Classification: Pharmacokinetics/Dynamics Study; Intervention Model: Crossover Assignment; Masking: Open Label; Primary Purpose: Treatment
NCT ID: NCT00915343
Other IDs: EudraCT: 2006-0007084-89, 104-07
First Received Date: June 5, 2009
Start Date: August 2007
Completion Date: January 2009
Last Updated Date: June 5, 2009
Last Verified Date: June 2009
Acronym: DC 06/02
Primary Completion Date: July 2008
Outcome Measures: To compare bioavailability between a once-daily modified release hydrocortisone oral tablet and a conventional thrice-daily replacement therapy in patients with chronic primary adrenal insufficiency.; To compare safety, tolerability and efficacy of the novel modified release formulation to the conventional thrice-daily replacement therapy.; To assess the safety of using the novel modified release formulation as "rescue therapy" during minor intercurrent illnesses in patients with primary adrenal insufficiency.; To assess long-term safety, tolerability and efficacy of the novel modified release formulation during glucocorticoid replacement therapy.
* * * * * * * * * * * *
9 * Unknown † *Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children
Condition: Adrenoleukodystrophy
Intervention: Procedure: blood sampling and medical records
Sponsors: Far Eastern Memorial Hospital; National Taiwan University Hospital; Fu Jen Catholic University
Gender: Both
Age Groups: Child / Adult / Senior
Phase:
Number Enrolled: 20
Funded By: Other
Study Type: Interventional
Study Design: Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Diagnostic
NCT ID: NCT00278044
Other IDs: 94028
First Received Date: January 16, 2006
Start Date: January 2006
Completion Date: July 2007
Last Updated Date: February 6, 2009
Last Verified Date: January 2006
Acronym:
Primary Completion Date:
Outcome Measure:
* * * * * * * * * * * *
10 * Recruiting * The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)
Conditions: X-linked Adrenoleukodystrophy; Adrenomyeloneuropathy
Intervention: Drug: Bezafibrate
Sponsors: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA); The Stop ALD Foundation
Gender: Male
Age Groups: Adult / Senior
Phase:
Number Enrolled: 10
Funded By: Other
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
NCT ID: NCT01165060
Other IDs: MEC 09/278
First Received Date: July 13, 2010
Start Date: July 2010
Completion Date: July 2011
Last Updated Date: July 16, 2010
Last Verified Date: July 2010
Acronym: BEZA
Primary Completion Date: July 2011
Outcome Measures: Very long chain fatty acids (VLCFA; C22:0, C24:0 and C26:0) in plasma, lymphocytes and erythrocytes.; Side effects; Cholesterol (total-HDL, and LDL-cholesterol) in plasma.
* * * * * * * * * * * *
11 * Completed * Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
Condition: Adrenoleukodystrophy
Interventions: Drug: glyceryl trierucate; Drug: glyceryl trioleate; Drug: interferon beta; Drug: thalidomide
Sponsors: FDA Office of Orphan Products Development; Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Gender: Male
Age Groups: Child / Adult / Senior
Phase:
Number Enrolled: 60
Funded By: U.S. Fed / Other
Study Type: Interventional
Study Design: Allocation: Randomized; Endpoint Classification: Efficacy Study; Masking: Double-Blind; Primary Purpose: Treatment
NCT ID: NCT00004450
Other IDs: 199/13532, KKI-94-06-16-01, KKI-FDR001052
First Received Date: October 18, 1999
Start Date: August 1998
Completion Date: November 2000
Last Updated Date: June 23, 2005
Last Verified Date: January 2001
Acronym:
Primary Completion Date:
Outcome Measure:
* * * * * * * * * * * *
12 * Unknown † * Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy
Condition: Adrenoleukodystrophy
Interventions: Drug: glyceryl trierucate; Drug: glyceryl trioleate
Sponsors: FDA Office of Orphan Products Development; Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Gender: Male
Group: Child
Phase:
Number Enrolled: 30
Funded By: U.S. Fed / Other
Study Type: Interventional
Study Design: Masking: Open Label; Primary Purpose: Treatment
NCT ID: NCT00004418
Other IDs: 199/13312, KKI-88-11-22-01, KKI-FDR000685
First Received Date: October 18, 1999
Start Date: April 1998
Completion Date:
Last Updated Date: June 15, 2009
Last Verified Date: September 2003
Acronym:
Primary Completion Date:
Outcome Measure:
* * * * * * * * * * * *
13 * Unknown † * Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Conditions: I Cell Disease; Fucosidosis; Globoid Cell Leukodystrophy; Adrenoleukodystrophy; Mannosidosis; Niemann-Pick Disease; Pulmonary Complications; Mucopolysaccharidosis I; Mucopolysaccharidosis VI; Metachromatic Leukodystrophy; Gaucher's Disease; Wolman Disease
Intervention:
Sponsor: Fairview University Medical Center
Gender: Both
Age Groups: Child / Adult / Senior
Phase:
Number Enrolled: 10
Funded By: Other
Study Type: Observational
Study Design: Primary Purpose: Screening
NCT ID: NCT00005900
Other IDs: 199/15111, UMN-MT-1999-18, UMN-MT-9818
First Received Date: June 2, 2000
Start Date: August 1999
Completion Date:
Last Updated Date: June 23, 2005
Last Verified Date: October 2003
Acronym:
Primary Completion Date:
Outcome Measure:
* * * * * * * * * * * *
14 * Completed * Study of Oral Cholic Acid in Patients With Inborn Errors of Bile Acid Synthesis
Conditions: Infantile Refsum's Disease; Zellweger Syndrome; Hyperpipecolic Acidemia; Adrenoleukodystrophy; Peroxisomal Disorders; Cholestasis; Bifunctional Enzyme Deficiency
Intervention:
Sponsors: National Center for Research Resources (NCRR); Children's Hospital Medical Center, Cincinnati
Gender: Both
Group: Child
Phase:
Number Enrolled: 21
Funded By: NIH / Other
Study Type: Observational
Study Design: Primary Purpose: Screening
NCT ID: NCT00007020
Other IDs: NCRR-M01RR08084-0009, CHMC-C-91-10-10
First Received Date: December 6, 2000
Start Date: August 2000
Completion Date:
Last Updated Date: June 23, 2005
Last Verified Date: December 2003
Acronym:
Primary Completion Date:
Outcome Measure:
* * * * * * * * * * * *
15 * Unknown † * Study of Bile Acids in Patients With Peroxisomal Disorders
Conditions: Infantile Refsum's Disease; Zellweger Syndrome; Bifunctional Enzyme Deficiency; Adrenoleukodystrophy
Interventions: Drug: chenodeoxycholic acid; Drug: cholic acid; Drug: ursodiol
Sponsors: FDA Office of Orphan Products Development; Children's Hospital Medical Center, Cincinnati
Gender: Both
Group: Child
Phase:
Number Enrolled: 25
Funded By: U.S. Fed / Other
Study Type: Interventional
Study Design: Primary Purpose: Treatment
NCT ID: NCT00004442
Other IDs: 199/13442, CHMC-C-FDR000995
First Received Date: October 18, 1999
Start Date:
Completion Date:
Last Updated Date: June 23, 2005
Last Verified Date: November 2000
Acronym:
Primary Completion Date:
Outcome Measure:
* * * * * * * * * * * *
16 * Recruiting * A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy
Conditions: Adrenomyeloneuropathy; Adrenoleukodystrophy
Intervention: Drug: Lorenzo's oil
Sponsors: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); Food and Drug Administration (FDA)
Gender: Both
Age Groups: Adult / Senior
Phase: Phase III
Number Enrolled: 240
Funded By: NIH / U.S. Fed
Study Type: Interventional
Study Design: Allocation: Randomized; Endpoint Classification: Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Investigator); Primary Purpose: Treatment
NCT ID: NCT00545597
Other IDs: R01 HD39276, R01FD003030
First Received Date: October 16, 2007
Start Date: March 2005
Completion Date: June 2012