Arimidex in McCune Albright Syndrome

AstraZeneca

Status and phase: Completed; Phase 2

Conditions: McCune-Albright Syndrome

Treatments: Drug: Arimidex 1 mg

Study type: Interventional

Funder types: Industry

Identifiers

NCT00055302

1033IL/0046

D5394C00046

Details and patient eligibility

About

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.

Enrollment: 40 patients

Sex: Female

Ages: Under 10 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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40 participants in 1 patient group

1

Experimental group

Treatment:

Drug: Arimidex 1 mg