Overview

This trial is active, not recruiting.

Conditions puberty, precocious, mccune-albright syndrome
Treatment fulvestrant
Phase phase 2
Sponsor AstraZeneca
Start date January 2006
End date December 2009
Trial size 34 participants
Trial identifier NCT00278915, D6992C00044, EUDRACT Number: 2005-004893-29

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 (early puberty) in girls with McCune-Albright syndrome (MAS).

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
fulvestrant Faslodex
intramuscular injection

Primary Outcomes

Measure
Change in the Frequency of Annualised Days of Vaginal Bleeding
time frame: 6 month pre-treatment observation period (baseline) followed by 12 month treatment period (on treatment period)

Secondary Outcomes

Measure
Percentage of Participants With Baseline Vaginal Bleeding Who Experienced ≥50% Reduction in the Number of Vaginal Bleeding Days
time frame: 6 month pre-treatment observation period (baseline) followed by 12 month treatment period (on treatment period)
Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over a 6 Month Trial Period.
time frame: 6 month pre-treatment observation period (baseline) followed by 12 month treatment period (on treatment period)
Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding .
time frame: 6 month pre-treatment observation period (baseline) followed by 12 month treatment period (on treatment period)
Change in Bone Age Advancement Over the First 6 Month Trial Period.
time frame: baseline to first 6 months of the treatment period
Change in Bone Age Advancement Over the Second 6 Month Trial Period.
time frame: baseline to second 6 months of the treatment period.
Change in Bone Age Advancement Over the Whole 12 Month Trial Period.
time frame: 6 month pre-treatment observation period (baseline) followed by 12 month treatment period (on treatment period)
Change in Growth Velocity (Annualised Growth Velocity i.e. cm/y) Over the First 6 Month Trial Period.
time frame: 6 month pre-treatment observation period (baseline) followed by 6 month treatment period (on treatment period)
Change in Growth Velocity (Annualised Growth Velocity i.e. cm/y) Over the Second 6 Month Trial Period.
time frame: 6 month pre-treatment observation period (baseline) followed by 12 month treatment period (on treatment period)
Change in Growth Velocity (Annualised Growth Velocity i.e. cm/y) Over the Whole 12 Month Trial Period.
time frame: 6 month pre-treatment observation period (baseline) followed by 12 month treatment period (on treatment period)
Change in Growth Velocity (Z-Score) Over the First 6 Month Trial Period.
time frame: 6 month pre-treatment observation period (baseline) followed by 6 month treatment period (on treatment period)
Change in Growth Velocity (Z-Score) Over the Second 6 Month Trial Period.
time frame: 6 month pre-treatment observation period (baseline) followed by 12 month treatment period (on treatment period)
Change in Growth Velocity (Z-Score) Over the Whole 12 Month Trial Period.
time frame: 6 month pre-treatment observation period (baseline) followed by 12 month treatment period (on treatment period)
Change in Uterine Volume From Baseline to Month 12 by Ultrasound.
time frame: Screening visit (baseline) and Month 12 during the treatment period.
Change in Uterine Volume From Baseline to Month 6 by Ultrasound.
time frame: Screening visit (baseline) and Month 6 during the treatment period.
Change in Uterine Volume From Month 6 to Month 12 by Ultrasound.
time frame: Month 6 and Month 12 during the treatment period.
Change in Ovarian Volume From Baseline to Month 12 by Ultrasound.
time frame: Screening visit (baseline), Months 6 and 12 during the treatment period.
Change in Ovarian Volume From Baseline to Month 6 by Ultrasound.
time frame: Screening visit (baseline), Months 6 and 12 during the treatment period.
Change in Ovarian Volume From Month 6 to Month 12 by Ultrasound.
time frame: Screening visit (baseline), Months 6 and 12 during the treatment period.
Hormone Assays: Serum Oestradiol.
time frame: Month 12 of the treatment period.
Hormone Assays: Luteinizing Hormone (LH).
time frame: Month 12 of the treatment period.
Hormone Assays: Follicle-stimulating Hormone (FSH).
time frame: Month 12 of the treatment period.
Hormone Assays: Testosterone.
time frame: Month 12 of the treatment period.
PK: Mean Clearance.
time frame: Throughout the 12 month treatment period.
PK: Mean Volume of Distribution (V1/F)
time frame: Throughout the 12 month treatment period.
PK: Mean Volume of Distribution (V2/F) .
time frame: Throughout the 12 month treatment period.
Change in Breast Tanner Stage From Baseline to Month 12.
time frame: 6 month pre-treatment observation period (result at Month 0 considered as baseline) followed by 12 month treatment period (on treatment period).
Change in Pubic Tanner Stage From Baseline to Month 12.
time frame: 6 month pre-treatment observation period (result at Month 0 considered as baseline) followed by 12 month treatment period (on treatment period).
Change in Predicted Adult Height (PAH) From Baseline to Month 12.
time frame: 6 month pre-treatment observation period (result at Screening considered as baseline) followed by 12 month treatment period (on treatment period).
Percentage of Patients With Gsα Mutation.
time frame: Screening assessment (baseline)

Eligibility Criteria

Female participants from 1 year up to 10 years old.

Inclusion Criteria: - Females less than or equal to 10 years of age (prior to 11th birthday) - Diagnosis of McCune-Albright syndrome (MAS) - Progressive precocious puberty (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

Additional Information

Official title An Open-label, Non-Comparative Trial to Evaluate the Safety, Efficacy and Pharmacokinetics of Faslodex (Fulvestrant) in Girls With Progressive Precocious Puberty Associated With McCune-Albright Syndrome
Trial information was received from ClinicalTrials.gov and was last updated in February 2016.
Information provided to ClinicalTrials.gov by AstraZeneca.