Faslodex in McCune-Albright Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 18 March 2024
• Main ID: NCT00278915
• Date of registration: 17/01/2006
• Prospective Registration: Yes
• Primary sponsor: AstraZeneca
• Public title: Faslodex in McCune-Albright Syndrome FMAS
• Scientific 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
• Date of first enrolment: January 31, 2006
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT00278915
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

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

Contacts

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

Key inclusion & exclusion criteria

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



Age minimum: 1 Year
Age maximum: 10 Years
Gender: Female

Health Condition(s) or Problem(s) studied

McCune-Albright Syndrome

Puberty, Precocious

Intervention(s)

Drug: Fulvestrant

Primary Outcome(s)

Change in Frequency of Annualized Days of Vaginal Bleeding on Treatment Compared to Baseline [Time Frame: Baseline (6 month pre-treatment observation period) through Month 12 treatment period]

Change in Growth Velocity (Annualized Growth Velocity in cm/Year) Over Whole 12-month Treatment Period Compared to Baseline [Time Frame: Baseline (6 month pre-treatment observation period) through Month 12 of treatment period]

Change in Growth Velocity (Z-score) Over the Whole 12-month Treatment Period Compared to Baseline [Time Frame: Baseline (6 month pre-treatment observation period) through Month 12 of treatment period]

Change in Rate of BA Advancement Over the Whole 12-month Treatment Period Compared to Baseline [Time Frame: Baseline (6-month pre-treatment observation period) through Month 12 of treatment period]

Change in Uterine Volume From Baseline to Month 6 as Assessed by Ultrasound [Time Frame: Baseline (pre-treatment baseline visit) and Month 6 of treatment period]

Change in Uterine Volume From Month 6 to Month 12/Final Visit as Assessed by Ultrasound [Time Frame: At Month 6 and Month 12/final visit treatment period]

Change Mean in Ovarian Volume From Month 6 to Month 12/Final Visit as Assessed by Ultrasound [Time Frame: At Month 6 and Month 12/final visit treatment period]

Hormone Assay: Serum Luteinizing Hormone (LH) Level [Time Frame: Month 12 (final visit) of treatment period]

Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over a 6-month Treatment Period [Time Frame: Baseline (6-month pre-treatment observation period) through Month 12 treatment period]

Change in Growth Velocity (Annualized Growth Velocity in cm/Year) Over First 6-month Treatment Period Compared to Baseline [Time Frame: Baseline (6 month pre-treatment observation period) through first 6-month of treatment period]

Change in Growth Velocity (Z-score) Over the Second 6-month Treatment Period Compared to Baseline [Time Frame: Baseline (6 month pre-treatment observation period) through second 6-month treatment period]

Change in Growth Velocity (Annualized Growth Velocity in cm/Year) Over Second 6-month Treatment Period Compared to Baseline [Time Frame: Baseline (6 month pre-treatment observation period) through second 6-month treatment period (ie, through 12-month treatment period)]

Change in Growth Velocity (Z-score) Over the First 6-month Treatment Period Compared to Baseline [Time Frame: Baseline (6 month pre-treatment observation period) through first 6-month treatment period]

Change in Mean Ovarian Volume From Baseline to Month 12/Final Visit as Assessed by Ultrasound [Time Frame: Baseline (pre-treatment baseline visit) and Month 12/final visit treatment period]

Change in Rate of Bone Age (BA) Advancement Over First 6-month Treatment Period Compared to Baseline [Time Frame: Baseline (6-month pre-treatment observation period) through Month 6 of treatment period]

Number of Participants With Compliance to Study Treatment [Time Frame: Day 1 through Month 12 of treatment period]

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) [Time Frame: Day 1 through 68.7 weeks (maximum observed duration)]

Percentage of Participants With Baseline Vaginal Bleeding Who Experienced = 50% Reduction in the Number of Vaginal Bleeding Days on Treatment Compared to Baseline [Time Frame: Baseline (6 month pre-treatment observation period) through Month 12 treatment period]

Change in Rate of BA Advancement Over Second 6-month Treatment Period Compared to Baseline [Time Frame: Baseline (6-month pre-treatment observation period) through second Month 6 of treatment period]

Hormone Assay: Serum Oestradiol Level [Time Frame: Month 12 (final visit) of treatment period]

Change in Mean Ovarian Volume From Baseline to Month 6 as Assessed by Ultrasound [Time Frame: Baseline (pre-treatment screening visit) and Month 6 of treatment period]

Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over the Whole 12-month Treatment Period [Time Frame: Baseline (6 month pre-treatment observation period) through Month 12 treatment period]

Change in Uterine Volume From Baseline to Month 12/Final Visit as Assessed by Ultrasound [Time Frame: Baseline (pre-treatment screening visit) and Month 12 treatment period]

Hormone Assay: Serum Follicle-stimulating Hormone (FSH) Level [Time Frame: Month 12 (final visit) of treatment period]

Hormone Assay: Serum Testosterone Level [Time Frame: Month 12 (final visit) of treatment period]

Mean Clearance of Fulvestrant [Time Frame: Post-dose: Weeks 1, 2, 3, and pre-dose: Week 4 of Month 1 for first 6 participants, then pre-dose steady state samples on 2 occasions between Months 6 and 12 with at least 1 month in between wherein first sample drawn at least 30 days following sixth dose]

Mean Volume of Distribution (V1/F) of Fulvestrant [Time Frame: Post-dose: Weeks 1, 2, 3, and pre-dose: Week 4 of Month 1 for first 6 participants, then pre-dose steady state samples on 2 occasions between Months 6 and 12 with at least 1 month in between wherein first sample drawn at least 30 days following sixth dose]

Mean Volume of Distribution (V2/F) of Fulvestrant [Time Frame: Post-dose: Weeks 1, 2, 3, and pre-dose: Week 4 of Month 1 for first 6 participants, then pre-dose steady state samples on 2 occasions between Months 6 and 12 with at least 1 month in between wherein first sample drawn at least 30 days following sixth dose]

Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs [Time Frame: Day 1 through 68.7 weeks (maximum observed duration)]

Number of Participants With Withdrawals From Study Treatment Due to TEAE [Time Frame: Day 1 through 68.7 weeks (maximum observed duration)]

Secondary Outcome(s)

Change in Tanner Stage of Breast From Baseline to Month 12/Final Visit [Time Frame: From Baseline (Month 0) through Month 12 treatment period]

Change in Predicted Adult Height (PAH) From Baseline to Month 12/Final Visit [Time Frame: From Baseline (screening visit) through Month 12 treatment period]

Change in Tanner Stage of Pubic Hair From Baseline to Month 12/Final Visit [Time Frame: From Baseline (Month 0) through Month 12 treatment period]

Percentage of Participants With McCune-Albright Syndrome (MAS) Associated G Protein a-subunit (Gsa) Mutation [Time Frame: Baseline (screening)]

Secondary ID(s)

2005-004893-26

D6992C00044

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 19/07/2011
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00278915