McCune-Albright Syndrome
Conditions
Keywords
vaginal bleeding, advanced bone age, fibrous dysplasia, MAS, McCune-Albright Syndrome
Brief summary
The primary objective of this study is to evaluate the safety and efficacy of anastrozole 1 mg given once daily in subjects with McCune-Albright Syndrome.
Interventions
DRUGArimidex 1 mg
Arimidex (anastrozole) 1mg once daily by mouth
Sponsors
AstraZeneca
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Age
No minimum to 10 Years
Healthy volunteers
No
Inclusion criteria
* informed written consent of parent/legal guardian and subject assent (as needed by local requirements) * females less than or equal to 10 years of age * diagnosed with McCune-Albright Syndrome * have progressive precocious puberty
Exclusion criteria
Any one of the following is regarded as a criterion for exclusion from the study: * any prior treatment of MAS associated with progressive precocious puberty with a third generation aromatase inhibitor (anastrozole, letrozole, exemestane) in which no clinical response was seen * concomitant treatment of precocious puberty associated with MAS, with the exception of bisphosphonates for polyostotic fibrous dysplasia and LHRH analogues in the case of central precocious puberty * liver function tests at screening visit (AST, ALT) \> or = 3x the upper limit of the reference range for age * known hypersensitivity to any component of study medication
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The efficacy of study treatment will be assessed based on the change from baseline measurements relating to vaginal bleeding, bone age, and growth velocity | 12 months or until the subject demonstrates the lack of efficacy bases upon progression of primary endpoints or experiences serious drug-related toxicity requiring withdrawal |
Countries
France, Germany, Italy, United Kingdom
Outcome results
None listed