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

Hide Participant Flow

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

Hide Baseline Characteristics

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) ]

Hide Outcome Measure 11

Measure Type	Secondary
Measure Title	Change in Growth Velocity (Z-Score) Over the First 6 Month Trial Period.
Measure Description	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.
Time Frame	6 month pre-treatment observation period (baseline) followed by 6 month treatment period (on treatment period)
Safety Issue	No

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)

Measured Values

	Fulvestrant
Number of Participants Analyzed [units: participants]	30
Change in Growth Velocity (Z-Score) Over the First 6 Month Trial Period. [units: Z-Score] Mean ± Standard Deviation	-1.60 ± 4.616

No statistical analysis provided for Change in Growth Velocity (Z-Score) Over the First 6 Month Trial 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) ]

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

Show Serious Adverse Events

Other Adverse Events

Show Other Adverse Events

More Information

Hide More Information

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:

	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.
	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.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: The PI cannot disclose or publish any Confidential Information to do with the trial without consent from AstraZeneca, and, for other information, the PI must withhold from publishing or presenting for 90 days if requested to do so by AstraZeneca.

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

To Top