Prospective, Open-Label, Multicenter, International Study of Mifepristone for Symptomatic Treatment of Cushing's Syndrome Caused by Ectopic Adrenal Corticotrophin Hormone (ACTH) Secretion
| Status: | Archived |
|---|---|
| Conditions: | Cardiology, Endocrine |
| Therapuetic Areas: | Cardiology / Vascular Diseases, Endocrinology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 7/1/2011 |
This study will evaluate whether the drug mifepristone can improve the symptoms of Cushing's
syndrome in people with ectopic adrenal corticotrophin hormone (ACTH) secretion. Cushing's
syndrome occurs when the adrenal glands produce too much cortisol, a hormone that helps to
regulate the body's use of salt and food. Excessive cortisol is usually the result of too
much ACTH, the hormone that causes the adrenal glands to make cortisol. The extra ACTH is
made either by a tumor in the pituitary gland (called Cushing's disease) or by a tumor
somewhere else (called ectopic ACTH secretion). Mifepristone blocks the action of cortisol
in the body. The drug has been used safely to treat a few people with Cushing's syndrome and
patients with certain kinds of cancer, gynecological diseases and psychiatric disorders.
People between 18 and 85 years of age with Cushing's syndrome caused by EXCESS ACTH
secretion may be eligible for this study. Candidates are admitted to the hospital for
evaluation to confirm Cushing's syndrome and to determine its cause. The evaluation includes
blood and urine tests, imaging tests, dexamethasone and corticotropin-releasing hormone
tests and inferior petrosal sinus sampling. Patients determined to have Cushing's syndrome
due to ECTOPIC ACTH secretion undergo imaging studies (CT, MRI and a nuclear medicine scan)
and begin mifepristone therapy.
Participants remain in the hospital for the following tests and procedures:
- Physical examination, electrocardiogram (EKG) and blood and urine tests
- Completion of medical questionnaires
- DEXA scan to determine bone mineral density and body composition
- Glucose tolerance test
- Urine pregnancy test and ultrasound to measure uterine lining thickness (for women)
Patients take mifepristone by mouth 3 times a day. The dose is increased every week or so
until symptoms improve or the highest dosage allowed is reached. Patients may remain in the
hospital for all or part of the dose-finding part of the study. During this period (usually
2 to 4 weeks), blood pressure, glucose tolerance and blood chemistries are measured and EKG
and urinalysis done every 5 to 14 days. When the mifepristone dose is stable patients remain
on that dose for at least 2 weeks and are then re-evaluated. Patients then return to the
hospital for evaluations every 3 months. Those who do well on the drug may continue to take
it for up to 12 months.
Between 10% and 20% of patients with hypercortisolism (Cushing's Syndrome) have tumoral
ectopic production of adrenocorticotropin hormone (ACTH) that causes cortisol excess. If an
ectopic tumor cannot be found or if surgery cannot be done, the treatment options include
medicines that reduce cortisol production and bilateral adrenalectomy. The available
medications that reduce cortisol production have important adverse effects and are not
effective in some patients and adrenalectomy leads to lifelong requirements for medical
hormone replacement. Thus, additional treatment options would be welcome. This study
evaluates a potential new medication for the treatment of these patients; mifepristone
blocks the effects of cortisol rather than decreasing its production. The purpose of this
study is to see whether this agent can improve diabetes or other symptoms of Cushing's
syndrome in subjects with ectopic ACTH secretion. Another purpose is to evaluate adverse
effects with this drug. Patients with presumed ectopic ACTH secretion and diabetes will
take mifepristone 600 mg daily by mouth, and the effect on diabetes and other symptoms of
Cushing's syndrome will be measured. Subjects will return to the hospital at 2, 3, 6, 9, and
12 months after starting mifepristione for evaluation of diabetes and other symptoms. The
agent will be available for up to 12 months for patients in whom it is effective.
Patients take mifepristone by mouth 3 times a day. Each dose will contain 200 mg. Patients
may remain in the hospital for all or part of the initial safety studies, every two weeks
for eight weeks. During this period blood pressure, glucose tolerance and blood chemistries
are measured and EKG and urinalysis done every two weeks. The mifepristone dose can be
decreased or stopped if there are adverse effects. When the mifepristone dose is stable for
eight weeks, patients will be re-evaluated. Patients then return to the hospital for
evaluations one month later and then every 3 months. Those who do well on the drug may
continue to take it for up to 12 months.
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
301-496-2563
National Institutes of Health Clinical Center The National Institutes of Health (NIH) Clinical Center in...
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