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Faslodex in McCune Albright Syndrome (FMAS)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
AstraZeneca
ClinicalTrials.gov Identifier:
NCT00278915
First received: January 17, 2006
Last updated: October 7, 2014
Last verified: October 2014

January 17, 2006
October 7, 2014
January 2006
December 2009   (final data collection date for primary outcome 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) ] [ Designated as safety issue: No ]
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.
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.
Complete list of historical versions of study NCT00278915 on ClinicalTrials.gov Archive Site
  • 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) ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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. ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    Change from pre-treatment period to the first 6 months of the treatment period. Z-score is [(growth velocity from the previous visit to the current visit - mean) / standard deviation(SD)], where the mean and SD are from the National Center for Health Statistics, Fels study.
  • 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) ] [ Designated as safety issue: No ]
    Change in growth velocity (Z-Score) from pre-treatment to the second 6 months of the treatment period. Z-score is [(growth velocity from the previous visit to the current visit - mean) / standard deviation(SD)], where the mean and SD are from the National Center for Health Statistics, Fels study.
  • 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) ] [ Designated as safety issue: No ]
    Change in growth velocity (Z-Score) from pre-treatment to the full 12 month treatment period. Z-score is [(growth velocity from the previous visit to the current visit - mean) / standard deviation(SD)], where the mean and SD are from the National Center for Health Statistics, Fels study.
  • Change in Uterine Volume From Baseline to Month 12 by Ultrasound. [ Time Frame: Screening visit (baseline) and Month 12 during the treatment period. ] [ Designated as safety issue: No ]
    Uterine volume was calculated via ultrasound using the formula: 0.5(longitudinal multiplied by anteroposterior multiplied by transverse), if all 3 linear dimensions were recorded. If all 3 linear dimensions were not recorded, uterine volume was not calculated.
  • Change in Uterine Volume From Baseline to Month 6 by Ultrasound. [ Time Frame: Screening visit (baseline) and Month 6 during the treatment period. ] [ Designated as safety issue: No ]
    Uterine volume was calculated via ultrasound using the formula: 0.5(longitudinal multiplied by anteroposterior multiplied by transverse), if all 3 linear dimensions were recorded. If all 3 linear dimensions were not recorded, uterine volume was not calculated.
  • Change in Uterine Volume From Month 6 to Month 12 by Ultrasound. [ Time Frame: Month 6 and Month 12 during the treatment period. ] [ Designated as safety issue: No ]
    Uterine volume was calculated via ultrasound using the formula: 0.5(longitudinal multiplied by anteroposterior multiplied by transverse), if all 3 linear dimensions were recorded. If all 3 linear dimensions were not recorded, uterine volume was not calculated.
  • Change in Ovarian Volume From Baseline to Month 12 by Ultrasound. [ Time Frame: Screening visit (baseline), Months 6 and 12 during the treatment period. ] [ Designated as safety issue: No ]
  • Change in Ovarian Volume From Baseline to Month 6 by Ultrasound. [ Time Frame: Screening visit (baseline), Months 6 and 12 during the treatment period. ] [ Designated as safety issue: No ]
  • 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. ] [ Designated as safety issue: No ]
  • Hormone Assays: Serum Oestradiol. [ Time Frame: Month 12 of the treatment period. ] [ Designated as safety issue: No ]
    Hormone assays: serum oestradiol at Screening visit (baseline), Month 12 of the treatment period. Results presented relate to Month 12 of the treatment period.
  • Hormone Assays: Luteinizing Hormone (LH). [ Time Frame: Month 12 of the treatment period. ] [ Designated as safety issue: No ]
    Hormone assays: Luteinizing hormone (LH) at Screening visit (baseline), Month 12 of the treatment period. Results presented relate to Month 12 of the treatment period.
  • Hormone Assays: Follicle-stimulating Hormone (FSH). [ Time Frame: Month 12 of the treatment period. ] [ Designated as safety issue: No ]
    Hormone assays: follicle-stimulating hormone (FSH)at Screening visit (baseline), Month 12 of the treatment period. Results presented relate to Month 12 of the treatment period..
  • Hormone Assays: Testosterone. [ Time Frame: Month 12 of the treatment period. ] [ Designated as safety issue: No ]
    Hormone assays: testosterone at Screening visit (baseline), Month 12 of the treatment period. Results presented relate to Month 12 of the treatment period..
  • PK: Mean Clearance. [ Time Frame: Throughout the 12 month treatment period. ] [ Designated as safety issue: No ]
    Mean clearance is the average amount of Fulvestrant which is eliminated
  • PK: Mean Volume of Distribution (V1/F) [ Time Frame: Throughout the 12 month treatment period. ] [ Designated as safety issue: No ]
    Total apparent volume of distribution (Vss/F) is the total apparent volume in the body into which Fulvestrant distributes at equilibrium. Vss/F = V1/F + V2/F. V1/F is the volume of the 1st compartment and V2/F is the volume of the second compartment. V1/F only is presented here. The measure of variability presented is the inter-individual error.
  • PK: Mean Volume of Distribution (V2/F) . [ Time Frame: Throughout the 12 month treatment period. ] [ Designated as safety issue: No ]
    Total apparent volume of distribution (Vss/F) is the total apparent volume in the body into which Fulvestrant distributes at equilibrium. Vss/F = V1/F + V2/F. V1/F is the volume of the 1st compartment and V2/F is the volume of the second compartment. V2/F only is presented here. The measure of variability presented is the inter-individual error.
  • 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). ] [ Designated as safety issue: No ]
    Change in breast Tanner stage from baseline to Month 12/last visit. Tanner stage (breast) is a score of range 1-5 where 1=no development and 5=adult breast
  • 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). ] [ Designated as safety issue: No ]
    Change in pubic Tanner stage from baseline to Month 12/last visit. Tanner stage (pubic) is a score of range 1-5 where 1=no development and 5=adult pubic hair
  • 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). ] [ Designated as safety issue: No ]
    Change in PAH from baseline to Month 12/final visit for patients equal to or over 6 years of age.
  • Percentage of Patients With Gsα Mutation. [ Time Frame: Screening assessment (baseline) ] [ Designated as safety issue: No ]
    McCune-Albright Syndrome(MAS) is caused by an activating mutation in the gene coding for the stimulatory subunit of the G protein, Gsα. The altered Gsα causes autonomous activation of G-protein stimulated cAMP formation, which in the gonads, results in episodic uncontrolled sex steroid production and subsequent pubertal development. For patients who provided separate specific informed consent, the percentage of patients with a Gsα mutation at screening was assessed by molecular analysis.
Change in (1) Tanner Stage of breast & pubic hair; and (2) Predicted adult height (PAH) for girls over the age of six.
Not Provided
Not Provided
 
Faslodex in McCune Albright Syndrome
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

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).

Not Provided
Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Puberty, Precocious
  • McCune-Albright Syndrome
Drug: Fulvestrant
intramuscular injection
Other Names:
  • Faslodex
  • ZD9238
Experimental: 1
Intervention: Drug: Fulvestrant
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
34
July 2023
December 2009   (final data collection date for primary outcome measure)

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
Female
1 Year to 10 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   France,   Germany,   Italy,   Russian Federation,   United Kingdom
 
NCT00278915
D6992C00044, EUDRACT Number: 2005-004893-29
Not Provided
AstraZeneca
AstraZeneca
Not Provided
Study Director: AstraZeneca Faslodex Medical Science Director, MD AstraZeneca
AstraZeneca
October 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP