Arimidex in McCune Albright Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 14 September 2015
• Main ID: NCT00055302
• Date of registration: 25/02/2003
• Prospective Registration: No
• Primary sponsor: AstraZeneca
• Public title: Arimidex in McCune Albright Syndrome
• Scientific title: An Open-label Study Evaluating the Safety and Efficacy of Anastrozole™ (ARIMIDEX) in the Treatment of Precocious Puberty in Girls With McCune-Albright Syndrome
• Date of first enrolment: August 2002
• Target sample size: 40
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00055302
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

France, Germany, Italy, Spain, United Kingdom, United States

Contacts

• Name: AstraZeneca Arimidex Medical Science Director, MD
• Affiliation: AstraZeneca

Key inclusion & 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



Age minimum: N/A
Age maximum: 10 Years
Gender: Female

Health Condition(s) or Problem(s) studied

McCune-Albright Syndrome

Intervention(s)

Drug: Arimidex 1 mg

Primary Outcome(s)

The efficacy of study treatment will be assessed based on the change from baseline measurements relating to vaginal bleeding, bone age, and growth velocity [Time Frame: 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]

Secondary ID(s)

D5394C00046

1033IL/0046

Source(s) of Monetary Support

Please refer to primary and secondary sponsors