CompletedPhase 2

Faslodex in McCune-Albright Syndrome

United States, France, Germany30 participantsStarted 2006-01-31

Plain-language 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)

Who can participate

Age range

1 Year – 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: * 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

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

Change in Frequency of Annualized Days of Vaginal Bleeding on Treatment Compared to Baseline

Timeframe: Baseline (6 month pre-treatment observation period) through Month 12 treatment period

Percentage of Participants With Baseline Vaginal Bleeding Who Experienced ≥ 50% Reduction in the Number of Vaginal Bleeding Days on Treatment Compared to Baseline

Timeframe: Baseline (6 month pre-treatment observation period) through Month 12 treatment period

Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over a 6-month Treatment Period

Timeframe: Baseline (6-month pre-treatment observation period) through Month 12 treatment period

Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over the Whole 12-month Treatment Period

Timeframe: Baseline (6 month pre-treatment observation period) through Month 12 treatment period

Change in Rate of Bone Age (BA) Advancement Over First 6-month Treatment Period Compared to Baseline

Timeframe: Baseline (6-month pre-treatment observation period) through Month 6 of treatment period

Trial details

NCT IDNCT00278915

SponsorAstraZeneca

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2009-12-08

Results submitted2010-12-06

View on ClinicalTrials.gov More Puberty, Precocious trials