Faslodex in McCune-Albright Syndrome (FMAS)

AstraZeneca

Status and phase: Completed; Phase 2

Conditions: McCune-Albright Syndrome; Puberty, Precocious

Treatments: Drug: Fulvestrant

Study type: Interventional

Funder types: Industry

Identifiers

NCT00278915

D6992C00044

2005-004893-26 (EudraCT Number)

Details and patient eligibility

About

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)

Enrollment: 30 patients

Sex: Female

Ages: 1 to 10 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Females less than or equal to 10 years of age (prior to 11th birthday)
• Diagnosis of MAS
• PPP associated with MAS

Exclusion criteria

• Received any prior treatment for PPP associated with MAS with fulvestrant
• Abnormal platelet count or liver function tests
• Bleeding disorders
• Long term anticoagulation therapy
• Known hypersensitivity to any component of the study drug

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

Fulvestrant

Experimental group

Description:

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.

Treatment:

Drug: Fulvestrant