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
Results First Received: December 6, 2010  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Puberty, Precocious
McCune-Albright Syndrome
Intervention: Drug: Fulvestrant

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Fulvestrant Fulvestrant (4 mg / kg)

Participant Flow:   Overall Study
    Fulvestrant  
STARTED     30 [1]
COMPLETED     29  
NOT COMPLETED     1  
Lack of Efficacy                 1  
[1] First 6 patients were dosed at 2mg/kg then increased to 4 mg/kg. Remaining 24 were dosed at 4mg/kg.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Fulvestrant Fulvestrant (4 mg / kg)

Baseline Measures
    Fulvestrant  
Number of Participants  
[units: participants]
  30  
Age  
[units: Years]
Mean ± Standard Deviation
  5.86  ± 1.846  
Gender  
[units: Participants]
 
Female     30  
Male     0  



  Outcome Measures
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1.  Primary:   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) ]

2.  Secondary:   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) ]

3.  Secondary:   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) ]

4.  Secondary:   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) ]

5.  Secondary:   Change in Bone Age Advancement Over the First 6 Month Trial Period.   [ Time Frame: baseline to first 6 months of the treatment period ]

6.  Secondary:   Change in Bone Age Advancement Over the Second 6 Month Trial Period.   [ Time Frame: baseline to second 6 months of the treatment period. ]

7.  Secondary:   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) ]

8.  Secondary:   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) ]

9.  Secondary:   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) ]

10.  Secondary:   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) ]

11.  Secondary:   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) ]

12.  Secondary:   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) ]

13.  Secondary:   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) ]

14.  Secondary:   Change in Uterine Volume From Baseline to Month 12 by Ultrasound.   [ Time Frame: Screening visit (baseline) and Month 12 during the treatment period. ]

15.  Secondary:   Change in Uterine Volume From Baseline to Month 6 by Ultrasound.   [ Time Frame: Screening visit (baseline) and Month 6 during the treatment period. ]

16.  Secondary:   Change in Uterine Volume From Month 6 to Month 12 by Ultrasound.   [ Time Frame: Month 6 and Month 12 during the treatment period. ]

17.  Secondary:   Change in Ovarian Volume From Baseline to Month 12 by Ultrasound.   [ Time Frame: Screening visit (baseline), Months 6 and 12 during the treatment period. ]

18.  Secondary:   Change in Ovarian Volume From Baseline to Month 6 by Ultrasound.   [ Time Frame: Screening visit (baseline), Months 6 and 12 during the treatment period. ]

19.  Secondary:   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. ]

20.  Secondary:   Hormone Assays: Serum Oestradiol.   [ Time Frame: Month 12 of the treatment period. ]

21.  Secondary:   Hormone Assays: Luteinizing Hormone (LH).   [ Time Frame: Month 12 of the treatment period. ]

22.  Secondary:   Hormone Assays: Follicle-stimulating Hormone (FSH).   [ Time Frame: Month 12 of the treatment period. ]

23.  Secondary:   Hormone Assays: Testosterone.   [ Time Frame: Month 12 of the treatment period. ]

24.  Secondary:   PK: Mean Clearance.   [ Time Frame: Throughout the 12 month treatment period. ]

25.  Secondary:   PK: Mean Volume of Distribution (V1/F)   [ Time Frame: Throughout the 12 month treatment period. ]

26.  Secondary:   PK: Mean Volume of Distribution (V2/F) .   [ Time Frame: Throughout the 12 month treatment period. ]

27.  Secondary:   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). ]

28.  Secondary:   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). ]

29.  Secondary:   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). ]

30.  Secondary:   Percentage of Patients With Gsα Mutation.   [ Time Frame: Screening assessment (baseline) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Gerard Lynch
Organization: AstraZeneca
e-mail: aztrial_results_posting@astrazeneca.com


No publications provided


Responsible Party: AstraZeneca
ClinicalTrials.gov Identifier: NCT00278915     History of Changes
Other Study ID Numbers: D6992C00044, EUDRACT Number: 2005-004893-29
Study First Received: January 17, 2006
Results First Received: December 6, 2010
Last Updated: October 7, 2014
Health Authority: United States: Food and Drug Administration