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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: September 24, 2012
Last verified: September 2012
History of Changes
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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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
  Show All Outcome Measures

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) ]
  Show Outcome Measure 1

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) ]
  Show Outcome Measure 2

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) ]
  Show Outcome Measure 3

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) ]
  Show Outcome Measure 4

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 ]
  Show Outcome Measure 5

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. ]
  Show Outcome Measure 6

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) ]
  Show Outcome Measure 7

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) ]
  Show Outcome Measure 8

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) ]
  Show Outcome Measure 9

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) ]
  Show Outcome Measure 10

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) ]
  Show Outcome Measure 11

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) ]
  Show Outcome Measure 12

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) ]
  Show Outcome Measure 13

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. ]
  Show Outcome Measure 14

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. ]
  Show Outcome Measure 15

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. ]
  Show Outcome Measure 16

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. ]
  Show Outcome Measure 17

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. ]
  Show Outcome Measure 18

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. ]
  Show Outcome Measure 19

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

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

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

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

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

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

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

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). ]
  Show Outcome Measure 27

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). ]
  Show Outcome Measure 28

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). ]
  Show Outcome Measure 29

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


  Serious Adverse Events
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  Other Adverse Events
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Time Frame No text entered.
Additional Description No text entered.

Frequency Threshold
Threshold above which other adverse events are reported   5%  

Reporting Groups
  Description
Fulvestrant Fulvestrant (4 mg / kg)

Other Adverse Events
    Fulvestrant  
Total, other (not including serious) adverse events    
# participants affected / at risk     25/30  
Ear and labyrinth disorders    
Ear Pain † 1  
# participants affected / at risk     4/30 (13.33%)  
Gastrointestinal disorders    
Abdominal Pain † 1  
# participants affected / at risk     8/30 (26.67%)  
Vomiting † 1  
# participants affected / at risk     8/30 (26.67%)  
Diarrhoea † 1  
# participants affected / at risk     5/30 (16.67%)  
Abdominal Pain Upper † 1  
# participants affected / at risk     4/30 (13.33%)  
Toothache † 1  
# participants affected / at risk     3/30 (10.00%)  
Nausea † 1  
# participants affected / at risk     2/30 (6.67%)  
General disorders    
Pyrexia † 1  
# participants affected / at risk     14/30 (46.67%)  
Injection Site Inflammation † 1  
# participants affected / at risk     4/30 (13.33%)  
Fatigue † 1  
# participants affected / at risk     2/30 (6.67%)  
Injection Site Pain † 1  
# participants affected / at risk     2/30 (6.67%)  
Infections and infestations    
Rhinitis † 1  
# participants affected / at risk     6/30 (20.00%)  
Bronchitis † 1  
# participants affected / at risk     2/30 (6.67%)  
Ear Infection † 1  
# participants affected / at risk     3/30 (10.00%)  
Gastroenteritis † 1  
# participants affected / at risk     3/30 (10.00%)  
Otitis Media † 1  
# participants affected / at risk     2/30 (6.67%)  
Pharyngitis † 1  
# participants affected / at risk     2/30 (6.67%)  
Pharyngitis Streptococcal † 1  
# participants affected / at risk     2/30 (6.67%)  
Sinusitis † 1  
# participants affected / at risk     2/30 (6.67%)  
Swine Influenza † 1  
# participants affected / at risk     2/30 (6.67%)  
Tonsillitis † 1  
# participants affected / at risk     3/30 (10.00%)  
Upper Respiratory Tract Infection † 1  
# participants affected / at risk     5/30 (16.67%)  
Vaginal Infection † 1  
# participants affected / at risk     2/30 (6.67%)  
Varicella † 1  
# participants affected / at risk     2/30 (6.67%)  
Urinary Tract Infection † 1  
# participants affected / at risk     3/30 (10.00%)  
Metabolism and nutrition disorders    
Decreased Appetite † 1  
# participants affected / at risk     3/30 (10.00%)  
Musculoskeletal and connective tissue disorders    
Pain In Extremity † 1  
# participants affected / at risk     5/30 (16.67%)  
Arthralgia † 1  
# participants affected / at risk     2/30 (6.67%)  
Bone Pain † 1  
# participants affected / at risk     2/30 (6.67%)  
Neck Pain † 1  
# participants affected / at risk     2/30 (6.67%)  
Nervous system disorders    
Headache † 1  
# participants affected / at risk     8/30 (26.67%)  
Lethargy † 1  
# participants affected / at risk     2/30 (6.67%)  
Respiratory, thoracic and mediastinal disorders    
Cough † 1  
# participants affected / at risk     7/30 (23.33%)  
Oropharyngeal Pain † 1  
# participants affected / at risk     4/30 (13.33%)  
Productive Cough † 1  
# participants affected / at risk     2/30 (6.67%)  
Skin and subcutaneous tissue disorders    
Rash † 1  
# participants affected / at risk     3/30 (10.00%)  
Eczema † 1  
# participants affected / at risk     2/30 (6.67%)  
Vascular disorders    
Hot Flush † 1  
# participants affected / at risk     2/30 (6.67%)  
† Events were collected by systematic assessment
1 Term from vocabulary, MedDRA 12.1



  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.


Limitations and Caveats
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
No text entered.  


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: September 24, 2012
Health Authority: United States: Food and Drug Administration