CompletedPhase 2

Arimidex in McCune Albright Syndrome

France, Germany, Italy40 participantsStarted 2002-08

Plain-language 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.

Who can participate

Age range

10 Years

Sex

FEMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: 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

Trial details

NCT IDNCT00055302

SponsorAstraZeneca

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2006-02

View on ClinicalTrials.gov More McCune-Albright Syndrome trials