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A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome Clinical Trials Resources presented on Clinical Trials Search is not meant to be a substitute for proven health advice, calls or treatment with a real medical. We aren't mDs. Always consult your doctor on A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome conditions. Clinical Trials Search.org is a website dedicated to listing clinical research studies in human subjects. A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome Clinical research trials and A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome healthcare trials take place in a lot of of localities throughout the U.S.. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials typically assess the effectiveness of new does drugs. The function of the studies / projects is to figure out specific human medical questions. Clinical trials are a popular means for doctors, government agencies, and private sector corporations to find cures for all varieties of conditions, like A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome. A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome Clinical Trials and other clinical trials allow volunteers to access health treatment options before they are available to the masses. Many times the subjects receive professional assistance for free, and every now and again they are compensated for their time. Sometimes there is a cost for a A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome clinical trial. Human subjects often obtain the finest healthcare possible for their A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome condition. Hazards are a reality, nevertheless, and might include additional or frequent dr. calls, health hazards (potentially life-jeopardizing), and/or the treatment being uneffective. Trials are federally regulated with stern guidelines to protect clinical trials patients.
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Home > "A" Clinical Trials Conditions > A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome  A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome
A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome
For Condition: Polyostotic Fibrous Dysplasia,McCune Albright Syndrome
Status: No longer recruiting
Sponsor(s): National Institute of Dental and Craniofacial Research (NIDCR) ,
Synopsis: This study will examine the effect of pegvisomant on growth hormone excess in patients with McCune-Albright syndrome (MAS). Patients with this disease have polyostotic fibrous dysplasia-a condition in which areas of normal bone are replaced with fibrous growth similar to scar tissue, abnormal skin pigmentation (birth marks) and precocious (early) puberty. About 10 percent of patients have excess growth hormone (GH). GH stimulates the production of another hormone called insulin-like growth factor 1 (IGF-1). Together, GH and IGF-1 affect bone growth. The excess of these hormones in MAS can cause overgrowth of the bones of the face, hands and feet, excess sweating, or increased height. Pegvisomant is a synthetic drug that binds to cell receptors where GH would normally bind, thus preventing the naturally occurring hormone from stimulating IGF-1 and bone growth as it normally would. This study will see if pegvisomant will reduce blood levels of IGF-1 and mitigate the effects of growth hormone excess, including bone pain, bone turnover, hand and foot swelling and sweating, and abnormal levels of related hormones. Patients who were screened for polyostotic fibrous dysplasia and MAS under NIH protocol 98-D-0145 and were found to have MAS with excess growth hormone are eligible for this 36-week study. The screening protocol includes a history and physical examination, blood and urine tests, hearing, eye and dental examinations, pain and physical function evaluations, endocrine and bone screening tests, various bone imaging studies, including magnetic resonance imaging (MRI) and computed tomography (CT) scans and bone biopsy in patients over 6 years old. Participants in the current study will receive daily injections of either pegvisomant or placebo (an inactive substance) for 12 weeks, followed by a 6-week "washout" period with no drug. Then, patients who received placebo will be switched, or "crossed over," to receive pegvisomant for another 12 weeks, and those who received pegvisomant will receive placebo. This will be followed by another 6-week washout period. The drug and placebo will be injected under the skin, similar to insulin injections. Blood and urine tests will be done at the beginning of the study and repeated every 6 weeks until the study ends.
Details: McCune-Albright Syndrome (MAS) was originally described as the triad of polyostotic fibrous dysplasia of bone, cafe-au-lait skin pigmentation and precocious puberty. Other endocrine abnormalities have been identified in this disease. Growth hormone (GH) excess is associated with MAS and occurs in approximately 10% of the patients. Current therapies of MAS involve separate treatment for the bone and endocrine diseases. We propose to test the effectiveness of a novel GH receptor antagonist, pegvisomant at reducing the growth hormone excess in these patients. Secondarily we shall also assess the impact of pegvisomant therapy on the fibrous dysplastic bone lesions associated with the disease. The subjects will be patients with MAS and non-suppressible growth hormone as determined by standard oral glucose tolerant testing (OGTT) and an elevated insulin-like growth factor-1 (IGF-I). It will be a randomized, blinded crossover design. The primary and secondary measures of efficacy will be: the normalization of serum (IGF-I), a reduction in signs and symptoms of growth hormone excess, and a net change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3). The effect of pegvisomant on the fibrous dysplastic bone activity in these patients will be determined by a net change in the levels of bone turnover markers.
Eligibility:
Study Type: Interventional, Treatment, Efficacy
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: INCLUSION CRITERIA: Diagnosis of PFD/MAS as required in Protocol 98-D-0145 Growth hormone excess will be determined as a non-suppressible serum growth hormone by oral glucose tolerance test (OGTT). The OGTT parameter will be serum GH greater than 2.0 ng/ml at 60 minutes after an oral load of 75g glucose. Two consecutive and duplicate measurements of serum IGF-I level should be at least 1.3 times greater than the upper limit of normal (age and sex adjusted according to laboratory normal range).
Total Enrollment: 10
Location and Contact Information:
National Institute of Dental And Craniofacial Research (NIDCR)
Bethesda, Maryland, 20892
United States
Additional Information:
Study ID Numbers: 010197; 01-D-0197
Study Start Date: June 18, 2001
Record last reviewed: June 6, 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00017927
Other Mccune Albright Syndrome Studies:
1. Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome
2. Testolactone for the Treatment of Girls with LHRH Resistant Precocious Puberty
3. Alendronate to Treat Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
4. Studies on Abnormal Bone from Patients with Polyostotic Fibrous Dysplasia and McCune Albright Syndrome
5. Screening and Natural History of Patients with Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
Related Studies:
Other McCune Albright Syndrome Clinical Trials
Other Maryland Clinical Trials
Other Bethesda Clinical Trials
A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome
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