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

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

Hide 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) ]

Measure Type	Primary
Measure Title	Change in the Frequency of Annualised Days of Vaginal Bleeding
Measure Description	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.
Time Frame	6 month pre-treatment observation period (baseline) followed by 12 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 the Frequency of Annualised Days of Vaginal Bleeding [units: days per year] Median ( Full Range )	-3.6 ( -42 to 185 )

No statistical analysis provided for Change in the Frequency of Annualised Days of Vaginal Bleeding

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) ]

Measure Type	Secondary
Measure Title	Percentage of Participants With Baseline Vaginal Bleeding Who Experienced ≥50% Reduction in the Number of Vaginal Bleeding Days
Measure Description	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.
Time Frame	6 month pre-treatment observation period (baseline) followed by 12 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.
Number of participants (23) = number of eligible participants who have had bleeding during the 6 month baseline period

Reporting Groups

	Description
Fulvestrant	Fulvestrant (4 mg / kg)

Measured Values

	Fulvestrant
Number of Participants Analyzed [units: participants]	23
Percentage of Participants With Baseline Vaginal Bleeding Who Experienced ≥50% Reduction in the Number of Vaginal Bleeding Days [units: Percentage of Participants]	73.9

No statistical analysis provided for Percentage of Participants With Baseline Vaginal Bleeding Who Experienced ≥50% Reduction in the Number of Vaginal Bleeding Days

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) ]

Measure Type	Secondary
Measure Title	Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over a 6 Month Trial Period.
Measure Description	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.
Time Frame	6 month pre-treatment observation period (baseline) followed by 12 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.
This particular protocolled endpoint does not relate to all patients but only those who had baseline vaginal bleeding - N=23 rather than N=30.

Reporting Groups

	Description
Fulvestrant	Fulvestrant (4 mg / kg)

Measured Values

	Fulvestrant
Number of Participants Analyzed [units: participants]	23
Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over a 6 Month Trial Period. [units: Percentage of Participants]	78

No statistical analysis provided for Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over a 6 Month Trial 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) ]

Measure Type	Secondary
Measure Title	Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding .
Measure Description	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.
Time Frame	6 month pre-treatment observation period (baseline) followed by 12 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]	23
Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding . [units: Percentage of Participants]	35

No statistical analysis provided for Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding .

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 ]

Measure Type	Secondary
Measure Title	Change in Bone Age Advancement Over the First 6 Month Trial Period.
Measure Description	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.
Time Frame	baseline to first 6 months of the 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 Bone Age Advancement Over the First 6 Month Trial Period. [units: cm/year] Mean ± Standard Deviation	-0.83 ± 1.507

No statistical analysis provided for Change in Bone Age Advancement Over the First 6 Month Trial 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. ]

Measure Type	Secondary
Measure Title	Change in Bone Age Advancement Over the Second 6 Month Trial Period.
Measure Description	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.
Time Frame	baseline to second 6 months of the 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]	29
Change in Bone Age Advancement Over the Second 6 Month Trial Period. [units: cm/year] Mean ± Standard Deviation	-1.10 ± 1.383

No statistical analysis provided for Change in Bone Age Advancement Over the Second 6 Month Trial 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) ]

Measure Type	Secondary
Measure Title	Change in Bone Age Advancement Over the Whole 12 Month Trial Period.
Measure Description	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.
Time Frame	6 month pre-treatment observation period (baseline) followed by 12 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 Bone Age Advancement Over the Whole 12 Month Trial Period. [units: cm/year] Mean ± Standard Deviation	-0.93 ± 1.343

No statistical analysis provided for Change in Bone Age Advancement Over the Whole 12 Month Trial 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) ]

Measure Type	Secondary
Measure Title	Change in Growth Velocity (Annualised Growth Velocity i.e. cm/y) Over the First 6 Month Trial Period.
Measure Description	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)
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 (Annualised Growth Velocity i.e. cm/y) Over the First 6 Month Trial Period. [units: cm/year] Mean ± Standard Deviation	-1.7 ± 4.35

No statistical analysis provided for Change in Growth Velocity (Annualised Growth Velocity i.e. cm/y) Over the First 6 Month Trial 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) ]

Measure Type	Secondary
Measure Title	Change in Growth Velocity (Annualised Growth Velocity i.e. cm/y) Over the Second 6 Month Trial Period.
Measure Description	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)
Time Frame	6 month pre-treatment observation period (baseline) followed by 12 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 (Annualised Growth Velocity i.e. cm/y) Over the Second 6 Month Trial Period. [units: cm/year] Mean ± Standard Deviation	-0.8 ± 4.49

No statistical analysis provided for Change in Growth Velocity (Annualised Growth Velocity i.e. cm/y) Over the Second 6 Month Trial 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) ]

Measure Type	Secondary
Measure Title	Change in Growth Velocity (Annualised Growth Velocity i.e. cm/y) Over the Whole 12 Month Trial Period.
Measure Description	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)
Time Frame	6 month pre-treatment observation period (baseline) followed by 12 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 (Annualised Growth Velocity i.e. cm/y) Over the Whole 12 Month Trial Period. [units: cm/year] Mean ± Standard Deviation	-1.4 ± 3.69

No statistical analysis provided for Change in Growth Velocity (Annualised Growth Velocity i.e. cm/y) Over the Whole 12 Month Trial 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) ]

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) ]

Measure Type	Secondary
Measure Title	Change in Growth Velocity (Z-Score) Over the Second 6 Month Trial Period.
Measure Description	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.
Time Frame	6 month pre-treatment observation period (baseline) followed by 12 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 Second 6 Month Trial Period. [units: Z-score] Mean ± Standard Deviation	-0.64 ± 4.606

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

Measure Type	Secondary
Measure Title	Change in Growth Velocity (Z-Score) Over the Whole 12 Month Trial Period.
Measure Description	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.
Time Frame	6 month pre-treatment observation period (baseline) followed by 12 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 Whole 12 Month Trial Period. [units: Z-Score] Mean ± Standard Deviation	-1.14 ± 4.078

No statistical analysis provided for Change in Growth Velocity (Z-Score) Over the Whole 12 Month Trial 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. ]

Measure Type	Secondary
Measure Title	Change in Uterine Volume From Baseline to Month 12 by Ultrasound.
Measure Description	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.
Time Frame	Screening visit (baseline) and Month 12 during the 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]	27
Change in Uterine Volume From Baseline to Month 12 by Ultrasound. [units: cm^3] Median ( Full Range )	-2.44 ( -10.20 to 6.56 )

No statistical analysis provided for Change in Uterine Volume From Baseline to Month 12 by Ultrasound.

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. ]

Measure Type	Secondary
Measure Title	Change in Uterine Volume From Baseline to Month 6 by Ultrasound.
Measure Description	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.
Time Frame	Screening visit (baseline) and Month 6 during the 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]	29
Change in Uterine Volume From Baseline to Month 6 by Ultrasound. [units: cm^3] Median ( Full Range )	-1.10 ( -15.10 to 6.04 )

No statistical analysis provided for Change in Uterine Volume From Baseline to Month 6 by Ultrasound.

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. ]

Measure Type	Secondary
Measure Title	Change in Uterine Volume From Month 6 to Month 12 by Ultrasound.
Measure Description	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.
Time Frame	Month 6 and Month 12 during the 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]	28
Change in Uterine Volume From Month 6 to Month 12 by Ultrasound. [units: cm^3] Median ( Full Range )	-0.13 ( -11.84 to 4.48 )

No statistical analysis provided for Change in Uterine Volume From Month 6 to Month 12 by Ultrasound.

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. ]

Measure Type	Secondary
Measure Title	Change in Ovarian Volume From Baseline to Month 12 by Ultrasound.
Measure Description	No text entered.
Time Frame	Screening visit (baseline), Months 6 and 12 during the 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]	21
Change in Ovarian Volume From Baseline to Month 12 by Ultrasound. [units: cm^3] Median ( Full Range )	1.01 ( -22.25 to 10.36 )

No statistical analysis provided for Change in Ovarian Volume From Baseline to Month 12 by Ultrasound.

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. ]

Measure Type	Secondary
Measure Title	Change in Ovarian Volume From Baseline to Month 6 by Ultrasound.
Measure Description	No text entered.
Time Frame	Screening visit (baseline), Months 6 and 12 during the 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]	25
Change in Ovarian Volume From Baseline to Month 6 by Ultrasound. [units: cm^3] Median ( Full Range )	0.10 ( -27.62 to 7.96 )

No statistical analysis provided for Change in Ovarian Volume From Baseline to Month 6 by Ultrasound.

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. ]

Measure Type	Secondary
Measure Title	Change in Ovarian Volume From Month 6 to Month 12 by Ultrasound.
Measure Description	No text entered.
Time Frame	Screening visit (baseline), Months 6 and 12 during the 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]	24
Change in Ovarian Volume From Month 6 to Month 12 by Ultrasound. [units: cm^3] Median ( Full Range )	0.76 ( -4.08 to 9.97 )

No statistical analysis provided for Change in Ovarian Volume From Month 6 to Month 12 by Ultrasound.

20. Secondary:

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

Measure Type	Secondary
Measure Title	Hormone Assays: Serum Oestradiol.
Measure Description	Hormone assays: serum oestradiol at Screening visit (baseline), Month 12 of the treatment period. Results presented relate to Month 12 of the treatment period.
Time Frame	Month 12 of the 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]	26
Hormone Assays: Serum Oestradiol. [units: pmol/Litres] Mean ± Standard Deviation	25.95 ± 30.718

No statistical analysis provided for Hormone Assays: Serum Oestradiol.

21. Secondary:

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

Measure Type	Secondary
Measure Title	Hormone Assays: Luteinizing Hormone (LH).
Measure Description	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.
Time Frame	Month 12 of the 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]	28
Hormone Assays: Luteinizing Hormone (LH). [units: IU/Litres] Mean ± Standard Deviation	0.11 ± 0.042

No statistical analysis provided for Hormone Assays: Luteinizing Hormone (LH).

22. Secondary:

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

Measure Type	Secondary
Measure Title	Hormone Assays: Follicle-stimulating Hormone (FSH).
Measure Description	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..
Time Frame	Month 12 of the 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]	29
Hormone Assays: Follicle-stimulating Hormone (FSH). [units: IU/Litres] Mean ± Standard Deviation	1.13 ± 1.024

No statistical analysis provided for Hormone Assays: Follicle-stimulating Hormone (FSH).

23. Secondary:

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

Measure Type	Secondary
Measure Title	Hormone Assays: Testosterone.
Measure Description	Hormone assays: testosterone at Screening visit (baseline), Month 12 of the treatment period. Results presented relate to Month 12 of the treatment period..
Time Frame	Month 12 of the 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]	28
Hormone Assays: Testosterone. [units: nmol/Litres] Mean ± Standard Deviation	0.65 ± 0.273

No statistical analysis provided for Hormone Assays: Testosterone.

24. Secondary:

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

Measure Type	Secondary
Measure Title	PK: Mean Clearance.
Measure Description	Mean clearance is the average amount of Fulvestrant which is eliminated
Time Frame	Throughout the 12 month 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
PK: Mean Clearance. [units: Litres/hour] Mean ± Standard Deviation	38.4 ± 11.56

No statistical analysis provided for PK: Mean Clearance.

25. Secondary:

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

Measure Type	Secondary
Measure Title	PK: Mean Volume of Distribution (V1/F)
Measure Description	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.
Time Frame	Throughout the 12 month 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
PK: Mean Volume of Distribution (V1/F) [units: Litres] Mean ± Standard Error	33000 ± 0.492

No statistical analysis provided for PK: Mean Volume of Distribution (V1/F)

26. Secondary:

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

Measure Type	Secondary
Measure Title	PK: Mean Volume of Distribution (V2/F) .
Measure Description	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.
Time Frame	Throughout the 12 month 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
PK: Mean Volume of Distribution (V2/F) . [units: Litres] Mean ± Standard Error	32700 ± 0.296

No statistical analysis provided for PK: Mean Volume of Distribution (V2/F) .

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). ]

Measure Type	Secondary
Measure Title	Change in Breast Tanner Stage From Baseline to Month 12.
Measure Description	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
Time Frame	6 month pre-treatment observation period (result at Month 0 considered as baseline) followed by 12 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 Breast Tanner Stage From Baseline to Month 12. [units: units on a scale] Median ( Full Range )	0.0 ( -3 to 2 )

No statistical analysis provided for Change in Breast Tanner Stage From Baseline to Month 12.

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). ]

Measure Type	Secondary
Measure Title	Change in Pubic Tanner Stage From Baseline to Month 12.
Measure Description	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
Time Frame	6 month pre-treatment observation period (result at Month 0 considered as baseline) followed by 12 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 Pubic Tanner Stage From Baseline to Month 12. [units: units on a scale] Median ( Full Range )	0.0 ( -2 to 1 )

No statistical analysis provided for Change in Pubic Tanner Stage From Baseline to Month 12.

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). ]

Measure Type	Secondary
Measure Title	Change in Predicted Adult Height (PAH) From Baseline to Month 12.
Measure Description	Change in PAH from baseline to Month 12/final visit for patients equal to or over 6 years of age.
Time Frame	6 month pre-treatment observation period (result at Screening considered as baseline) followed by 12 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]	17
Change in Predicted Adult Height (PAH) From Baseline to Month 12. [units: cm] Mean ± Standard Deviation	0.5 ± 4.10

No statistical analysis provided for Change in Predicted Adult Height (PAH) From Baseline to Month 12.

30. Secondary:

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

Measure Type	Secondary
Measure Title	Percentage of Patients With Gsα Mutation.
Measure Description	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.
Time Frame	Screening assessment (baseline)
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
Percentage of Patients With Gsα Mutation. [units: Percentage of participants]	23

No statistical analysis provided for Percentage of Patients With Gsα Mutation.

Serious Adverse Events

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Other Adverse Events

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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:

	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

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