Conditions: Puberty, Precocious McCune-Albright Syndrome
Keywords: Progressive Precocious Puberty PPP McCune-Albright Syndrome MAS
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 30
Healthy volunteers: no

Brief summary:
The purpose of this study is to evaluate the safety, effectiveness and pharmacokinetics of a study drug called Faslodex (fulvestrant) in the treatment of progressive precocious puberty (PPP) (early puberty) in girls with McCune-Albright syndrome (MAS)

Detailed description:

Arms:
arm 1: Participants will receive intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants will be dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants will be dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.

Interventions: intervention 1: Fulvestrant
Intervention descriptions: intervention 1: Participants will receive intramuscular injection of fulvestrant as stated in arm description.

Protocol document: