Polyostotic Fibrous Dysplasia Clinical Trial
Official title:
A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome
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.
Status | Completed |
Enrollment | 10 |
Est. completion date | June 2005 |
Est. primary completion date | |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A and older |
Eligibility |
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). |
Endpoint Classification: Efficacy Study, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | National Institute of Dental And Craniofacial Research (NIDCR) | Bethesda | Maryland |
Lead Sponsor | Collaborator |
---|---|
National Institute of Dental and Craniofacial Research (NIDCR) |
United States,
Chanson P, Dib A, Visot A, Derome PJ. McCune-Albright syndrome and acromegaly: clinical studies and responses to treatment in five cases. Eur J Endocrinol. 1994 Sep;131(3):229-34. — View Citation
Mastorakos G, Mitsiades NS, Doufas AG, Koutras DA. Hyperthyroidism in McCune-Albright syndrome with a review of thyroid abnormalities sixty years after the first report. Thyroid. 1997 Jun;7(3):433-9. — View Citation
Shenker A, Weinstein LS, Moran A, Pescovitz OH, Charest NJ, Boney CM, Van Wyk JJ, Merino MJ, Feuillan PP, Spiegel AM. Severe endocrine and nonendocrine manifestations of the McCune-Albright syndrome associated with activating mutations of stimulatory G protein GS. J Pediatr. 1993 Oct;123(4):509-18. — View Citation
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