Clinical Trial: Faslodex in McCune Albright Syndrome

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

Brief 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).

Detailed Summary:
Sponsor: AstraZeneca

Current Primary Outcome: 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) ]

Change in the frequency of annualised days of vaginal bleeding during the 12 month treatment period compared to the 6 month baseline period, based on a worst-case scenario calculation .i.e. missing diary card days counted as bleeding days.


Original Primary Outcome: Change in annualized number of vaginal bleed days on treatment compared to baseline. Change in rate of increase in bone age and growth rate on treatment compared to baseline.

Current Secondary Outcome:

  • 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 ≥50% reduction in the number of vaginal bleeding days during the 12 month treatment period compared to the 6 month baseline 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 for at least 180 consecutive days during the 12 month treatment period, based on a worst-case approach i.e. missing diary card days counted as bleeding days.
  • 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) ]
    Percentage of participants with baseline vaginal bleeding who experienced cessation of vaginal bleeding for the full 12 month treatment period, based on a worst-case approach i.e. missing diary card days counted as bleeding days.
  • 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 the rate of increase in bone age from pre treatment (based on the 6 month retrospective visit) to the first 6 months of the treatment period. Bone age advancement for a particular time period was calculated as the increase in bone age over that time period adjusted (ie, normalized) for the length of that time 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 the rate of increase in bone age from pre treatment (based on the 6 month retrospective visit) to the second 6 months of the treatment period. Bone age advancement for a particular time period was calculated as the increase in bone age over that time period adjusted (ie, normalized) for the length of that time 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 the rate of increase in bone age from pre treatment (based on the 6 month retrospective visit) to the full 12 month treatment period. (Using last value carried forward method for the one patient who withdrew soon after their month 6 bone scan) Bone age advancement for a particular time period was calculated as the increase in bone age over that time period adjusted (ie, normalized) for the length of that time 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) from the pre treatment period to the first 6 months of the treatment period. Growth velocity for a particular time period was calculated as the increase in height over that time period divided by the length of that time period (expressed in cm/year)
  • 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) from the pre treatment period to the second 6 months of the treatment period. Growth velocity for a particular time period was calculated as the increase in height over that time period divided by the length of that time period (expressed in cm/year)
  • 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 (annualised growth velocity i.e. cm/y) from the pre treatment period to the full 12 month treatment period. Growth velocity for a particular time period was calculated as the increase in height over that time period divided by the length of that time period (expressed in cm/year)
  • 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 from pre-treatment period to the first 6 months of the treatment peri

    Original Secondary Outcome: Change in (1) Tanner Stage of breast & pubic hair; and (2) Predicted adult height (PAH) for girls over the age of six.

    Information By: AstraZeneca

    Dates:
    Date Received: January 17, 2006
    Date Started: January 2006
    Date Completion: July 2023
    Last Updated: February 14, 2017
    Last Verified: February 2017