Trial Outcomes & Findings for Faslodex in McCune-Albright Syndrome (NCT NCT00278915)

Last Updated: 2024-03-05

Results Overview

Vaginal bleeding days are defined as the number of days in which vaginal bleeding, (including spotting) occurred. In order to annualize, a 12-month period is defined as 360 days and a 6-month period is defined as 180 days. Frequency of annualized vaginal bleeding days = \[(number of vaginal bleeding days)/(total number of days of the time interval under consideration)\] multiplied by 360. Change in frequency is equal to the on-treatment frequency minus the baseline frequency. Diary cards will capture days of vaginal bleeding during the 12-month treatment period. Change in the frequency of annualized days of vaginal bleeding during the 12-month treatment period compared to the 6-month baseline period, based on a worst-case scenario calculation (ie, missing diary card days counted as bleeding days) are reported.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

Baseline (6 month pre-treatment observation period) through Month 12 treatment period

Results posted on

2024-03-05

Participant Flow

The study was conducted at study sites located in France, Germany, Italy, Russian Federation, United Kingdom, and the United States of America.

A total of 30 participants were enrolled in this study.

Participant milestones

Participant milestones
Measure	Fulvestrant Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Overall Study STARTED	30
Overall Study COMPLETED	29
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Fulvestrant Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Overall Study Disease progression	1

Baseline Characteristics

Faslodex in McCune-Albright Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Age, Continuous	5.86 Years STANDARD_DEVIATION 1.846 • n=5 Participants
Sex: Female, Male Female	30 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	23 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	26 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline (6 month pre-treatment observation period) through Month 12 treatment period

Population: The full-analysis set (FAS) population included participants who received at least 1 dose of study drug.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Frequency of Annualized Days of Vaginal Bleeding on Treatment Compared to Baseline	-3.6 Days per year Interval -42.0 to 185.0

PRIMARY outcome

Timeframe: Baseline (6 month pre-treatment observation period) through Month 12 treatment period

Population: The FAS population included participants who received at least 1 dose of study drug. Here, number of participants analyzed denotes those participants who had bleeding during the 6 month baseline period.

The percentage change in frequency is defined as 100% times the difference (the on-treatment period frequency minus the baseline period frequency), divided by the baseline period frequency. The percentage of participants with baseline vaginal bleeding days who experienced ≥ 50% reduction in the number of vaginal bleeding days during the 12 month treatment period compared to the 6 month baseline period based on a worst-case approach (ie, missing diary card days counted as bleeding days) are reported.

Outcome measures

Outcome measures
Measure	Fulvestrant n=23 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Percentage of Participants With Baseline Vaginal Bleeding Who Experienced ≥ 50% Reduction in the Number of Vaginal Bleeding Days on Treatment Compared to Baseline	73.9 Percentage of Participants Interval 51.6 to 89.8

PRIMARY outcome

Timeframe: Baseline (6-month pre-treatment observation period) through Month 12 treatment period

Percentage of participants with baseline vaginal bleeding who experienced cessation of vaginal bleeding days over a 6-month treatment period based on a worst-case approach (ie, missing diary card days counted as bleeding days) are reported.

Outcome measures

Outcome measures
Measure	Fulvestrant n=23 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over a 6-month Treatment Period	78.3 Percentage of Participants Interval 56.3 to 92.5

PRIMARY outcome

Timeframe: Baseline (6 month pre-treatment observation period) through Month 12 treatment period

Percentage of participants with baseline vaginal bleeding who experienced cessation of vaginal bleeding days over a 12-month treatment period based on a worst-case approach (ie, missing diary card days counted as bleeding days) are reported.

Outcome measures

Outcome measures
Measure	Fulvestrant n=23 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over the Whole 12-month Treatment Period	34.8 Percentage of Participants Interval 16.4 to 57.3

PRIMARY outcome

Timeframe: Baseline (6-month pre-treatment observation period) through Month 6 of treatment period

Population: The FAS population included participants who received at least 1 dose of study drug.

Change in rate of BA advancement over first 6-month treatment period compared to baseline (6-month pre-treatment observation period) is reported. Increase in BA is defined as BA (expressed as fractional years) at end of time period minus BA at beginning of time period (unit: years). Rate of increase in BA for a particular time interval is increase in BA during this time interval adjusted (ie, normalized) for the length of this time interval. Rate of BA advancement is change in BA (years) divided by change in chronological age (CA) (years). Change in rate of increase in BA from baseline period to on-treatment period is defined as increase in BA divided by change in CA (in fractional years) between BA radiograph dates. It is calculated as \[(BA6 - BA0)/(CA6 - CA0)\] - \[(BA0 - BA\*)/(CA0 - CA\*)\], where 6, 0, \* stand for first Month 6 Visit, Month 0 Visit, and the 6-month retrospective visit, respectively.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Rate of Bone Age (BA) Advancement Over First 6-month Treatment Period Compared to Baseline	-0.83 Ratio Standard Deviation 1.507

PRIMARY outcome

Timeframe: Baseline (6-month pre-treatment observation period) through second Month 6 of treatment period

Population: The FAS population included participants who received at least 1 dose of study drug. Here, number of participants analyzed denotes those participants who were evaluable at the specified time point.

Change in rate of BA advancement over second 6-month treatment period compared to baseline (6-month pre-treatment observation period) is reported. Increase in BA is defined as BA (expressed as fractional years) at end of time period minus BA at beginning of time period (unit: years). Rate of increase in BA for a particular time interval is increase in BA during this time interval adjusted (ie, normalized) for the length of this time interval. Rate of BA advancement is change in BA (years) divided by change in CA (years). Change in rate of increase in BA from baseline period to on-treatment period is defined as increase in BA divided by change in CA (in fractional years) between the BA radiograph dates. It is calculated as \[(BA6 - BA0)/(CA6 - CA0)\] - \[(BA0 - BA\*)/(CA0 - CA\*)\], where 6, 0, \* stand for second Month 6 Visit, Month 0 Visit, and the 6-month retrospective visit, respectively.

Outcome measures

Outcome measures
Measure	Fulvestrant n=29 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Rate of BA Advancement Over Second 6-month Treatment Period Compared to Baseline	-1.10 Ratio Standard Deviation 1.383

PRIMARY outcome

Timeframe: Baseline (6-month pre-treatment observation period) through Month 12 of treatment period

Population: The FAS population included participants who received at least 1 dose of study drug.

Change in rate of BA advancement over whole 12-month treatment period compared to baseline (6-month pre-treatment observation period) is reported. Increase in BA is defined as BA (expressed as fractional years) at end of time period minus BA at beginning of time period (unit: years). Rate of increase in BA for a particular time interval is increase in BA during this time interval adjusted (ie, normalized) for the length of this time interval. Rate of BA advancement is change in BA (years) divided by change in CA (years). Change in rate of increase in BA from baseline period to on-treatment period is defined as increase in BA divided by change in CA (in fractional years) between the BA radiograph dates. It is calculated as \[(BA12 - BA0) / (CA12 - CA0)\] - \[(BA0 - BA\*) / (CA0 - CA\*)\], where 12, 0, \* stand for Month 12 Visit, Month 0 Visit, and the 6-month retrospective visit, respectively.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Rate of BA Advancement Over the Whole 12-month Treatment Period Compared to Baseline	-0.93 Ratio Standard Deviation 1.343

PRIMARY outcome

Timeframe: Baseline (6 month pre-treatment observation period) through first 6-month of treatment period

Population: The FAS population included participants who received at least 1 dose of study drug.

Change in growth velocity (annualized growth velocity in cm/year) from the baseline (pre-treatment period) to first 6-month treatment period is reported. 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). Baseline growth velocity was calculated from 6-month observational/retrospective period of the study. Change in growth velocity was calculated as growth velocity on treatment minus change in growth velocity during baseline.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Growth Velocity (Annualized Growth Velocity in cm/Year) Over First 6-month Treatment Period Compared to Baseline	-1.7 cm/year Standard Deviation 4.35

PRIMARY outcome

Timeframe: Baseline (6 month pre-treatment observation period) through second 6-month treatment period (ie, through 12-month treatment period)

Population: The FAS population included participants who received at least 1 dose of study drug.

Change in growth velocity (annualized growth velocity in cm/year) from the baseline (pre-treatment period) to second 6-month treatment period is reported. 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). Baseline growth velocity was calculated from 6-month observational/retrospective period of the study. Change in growth velocity was calculated as growth velocity on treatment minus change in growth velocity during baseline.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Growth Velocity (Annualized Growth Velocity in cm/Year) Over Second 6-month Treatment Period Compared to Baseline	-0.8 cm/year Standard Deviation 4.49

PRIMARY outcome

Timeframe: Baseline (6 month pre-treatment observation period) through Month 12 of treatment period

Population: The FAS population included participants who received at least 1 dose of study drug.

Change in growth velocity (annualized growth velocity in cm/year) from the baseline (pre-treatment period) to the 12-month treatment period is reported. 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). Baseline growth velocity was calculated from 6-month observational/retrospective period of the study. Change in growth velocity was calculated as growth velocity on treatment minus change in growth velocity during baseline.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Growth Velocity (Annualized Growth Velocity in cm/Year) Over Whole 12-month Treatment Period Compared to Baseline	-1.4 cm/year Standard Deviation 3.69

PRIMARY outcome

Timeframe: Baseline (6 month pre-treatment observation period) through first 6-month treatment period

Population: The FAS population included participants who received at least 1 dose of study drug.

Change in growth velocity (Z-score) from baseline period to the first 6 months of treatment period is reported. The Z-score (also known as Standard Deviation Score \[SDS\]) is defined as \[(growth velocity from the previous visit to the current visit minus mean growth velocity) divided by standard deviation (SD)\], where the mean and SD are the age- and gender-specific statistics for growth velocity from the National Center for Health Statistics, Fels study and age is the age at the current visit. Baseline growth velocity was calculated from 6-month observational/retrospective period of the study. Z-score of 0 represents the population mean for growth velocity. For McCune-Albright Syndrome, Z-score below mean is a better outcome.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Growth Velocity (Z-score) Over the First 6-month Treatment Period Compared to Baseline	-1.60 Unit on a score Standard Deviation 4.616

PRIMARY outcome

Timeframe: Baseline (6 month pre-treatment observation period) through second 6-month treatment period

Population: The FAS population included participants who received at least 1 dose of study drug.

Change in growth velocity (Z-score) from baseline period to the second 6 months of treatment period is reported. The Z-score (also known as SDS) is defined as \[(growth velocity from the previous visit to the current visit minus mean growth velocity) divided by SD\], where the mean and SD are the age- and gender-specific statistics for growth velocity from the National Center for Health Statistics, Fels study and age is the age at the current visit. Baseline growth velocity was calculated from 6-month observational/retrospective period of the study. Z-score of 0 represents the population mean for growth velocity. For McCune-Albright Syndrome, Z-score below mean is a better outcome.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Growth Velocity (Z-score) Over the Second 6-month Treatment Period Compared to Baseline	-0.64 Unit on a score Standard Deviation 4.606

PRIMARY outcome

Timeframe: Baseline (6 month pre-treatment observation period) through Month 12 of treatment period

Population: The FAS population included participants who received at least 1 dose of study drug.

Change in growth velocity (Z-score) from baseline period to 12 months of treatment period is reported. The Z-score (also known as SDS) is defined as \[(growth velocity from the previous visit to the current visit minus mean growth velocity) divided by SD)\], where the mean and SD are the age- and gender-specific statistics for growth velocity from the National Center for Health Statistics, Fels study and age is the age at the current visit. Baseline growth velocity was calculated from 6-month observational/retrospective period of the study. Z-score of 0 represents the population mean for growth velocity. For McCune-Albright Syndrome, Z-score below mean is a better outcome.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Growth Velocity (Z-score) Over the Whole 12-month Treatment Period Compared to Baseline	-1.14 Unit on a score Standard Deviation 4.078

PRIMARY outcome

Timeframe: Baseline (pre-treatment baseline visit) and Month 6 of treatment period

Population: The FAS population included participants who received at least 1 dose of study drug. Here, number of participants analyzed denotes those participants with data at both time points.

Uterine volume was calculated via ultrasound using the formula: 0.5 multiplied by (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 was calculated as Month 6 volume (by ultrasound) minus screening visit volume (by ultrasound). Baseline (screening visit) is the pre-treatment baseline visit.

Outcome measures

Outcome measures
Measure	Fulvestrant n=29 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Uterine Volume From Baseline to Month 6 as Assessed by Ultrasound	-1.10 Cubic centimeters Interval -15.1 to 6.04

PRIMARY outcome

Timeframe: At Month 6 and Month 12/final visit treatment period

Population: The FAS population included participants who received at least 1 dose of study drug. Here, number of participants analyzed denotes those participants with data at both time points.

Uterine volume was calculated via ultrasound using the formula: 0.5 multiplied by (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/final visit was calculated as Month 12/finial visit volume (by ultrasound) minus Month 6 volume (by ultrasound).

Outcome measures

Outcome measures
Measure	Fulvestrant n=28 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Uterine Volume From Month 6 to Month 12/Final Visit as Assessed by Ultrasound	-0.13 Cubic centimetres Interval -11.84 to 4.48

PRIMARY outcome

Timeframe: Baseline (pre-treatment screening visit) and Month 12 treatment period

Population: The FAS population included participants who received at least 1 dose of study drug. Here, number of participants analyzed denotes those participants with data at both time points.

Uterine volume was calculated via ultrasound using the formula: 0.5 multiplied by (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 12/final visit was calculated as End of Study volume (by ultrasound) minus Screening Visit volume (by ultrasound). Baseline (screening visit) is the pre-treatment baseline visit.

Outcome measures

Outcome measures
Measure	Fulvestrant n=27 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Uterine Volume From Baseline to Month 12/Final Visit as Assessed by Ultrasound	-2.44 Cubic centimeters Interval -10.2 to 6.56

PRIMARY outcome

Timeframe: Baseline (pre-treatment screening visit) and Month 6 of treatment period

Population: The FAS population included participants who received at least 1 dose of study drug. Here, number of participants analyzed denotes those participants with data at both time points.

The mean ovarian volume was the average of both ovaries. Average volume was calculated as 0.5 multiplied by (volume of left ovary plus volume of right ovary) if both volumes were calculated; otherwise, average ovarian volume was considered missing. The volume of each ovary was calculated via ultrasound using the formula: 0.5 multiplied by longitudinal dimension multiplied by anterior-posterior dimension multiplied by transverse dimension. Change in mean ovarian volume from baseline to Month 6 was calculated as Month 6 mean volume minus Screening Visit mean volume. Baseline (screening visit) is the pre-treatment baseline visit.

Outcome measures

Outcome measures
Measure	Fulvestrant n=25 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Mean Ovarian Volume From Baseline to Month 6 as Assessed by Ultrasound	0.10 Cubic centimetres Interval -27.62 to 7.96

PRIMARY outcome

Timeframe: At Month 6 and Month 12/final visit treatment period

Population: The FAS population included participants who received at least 1 dose of study drug. Here, number of participants analyzed denotes those participants with data at both time points.

The mean ovarian volume was the average of both ovaries. Average volume was calculated as 0.5 multiplied by (volume of left ovary plus volume of right ovary) if both volumes were calculated; otherwise, average ovarian volume was considered missing. The volume of each ovary was calculated via ultrasound using the formula: 0.5 multiplied by longitudinal dimension multiplied by anterior-posterior dimension multiplied by transverse dimension. Change in mean ovarian volume from Month 6 to Month 12/final visit was calculated as Month 12/final visit mean volume minus Month 6 mean volume.

Outcome measures

Outcome measures
Measure	Fulvestrant n=24 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change Mean in Ovarian Volume From Month 6 to Month 12/Final Visit as Assessed by Ultrasound	0.76 Cubic centimetres Interval -4.08 to 9.97

PRIMARY outcome

Timeframe: Baseline (pre-treatment baseline visit) and Month 12/final visit treatment period

Population: The FAS population included participants who received at least 1 dose of study drug. Here, number of participants analyzed denotes those participants with data at both time points.

The mean ovarian volume was the average of both ovaries. Average ovarian volume was calculated as 0.5 multiplied by (volume of left ovary plus volume of right ovary) if both volumes were calculated; otherwise, average ovarian volume was considered missing. The volume of each ovary was calculated via ultrasound using the formula: 0.5 multiplied by longitudinal dimension multiplied by anterior-posterior dimension multiplied by transverse dimension. Change in mean ovarian volume from baseline to the end of the study was calculated as End of Study mean volume minus Screening Visit mean volume. Baseline (screening visit) is the pre-treatment baseline visit.

Outcome measures

Outcome measures
Measure	Fulvestrant n=21 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Mean Ovarian Volume From Baseline to Month 12/Final Visit as Assessed by Ultrasound	1.01 Cubic centimetres Interval -22.25 to 10.36

PRIMARY outcome

Timeframe: Post-dose: Weeks 1, 2, 3, and pre-dose: Week 4 of Month 1 for first 6 participants, then pre-dose steady state samples on 2 occasions between Months 6 and 12 with at least 1 month in between wherein first sample drawn at least 30 days following sixth dose

Population: Population pharmacokinetic (PK) analysis set included all participants who received at least 1 dose of study drug and have evaluable PK data.

Mean clearance of fulvestrant is reported.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Mean Clearance of Fulvestrant	38.4 Litres/hour Standard Deviation 11.56

PRIMARY outcome

Population: Population PK analysis set included all participants who received at least 1 dose of study drug and have evaluable PK data.

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 first compartment and V2/F is the volume of the second compartment. V1/F of fulvestrant is reported. The measure of variability presented is the inter-individual error.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Mean Volume of Distribution (V1/F) of Fulvestrant	33000 Litres Standard Error NA The measure of variability presented is the inter-individual error, not the Standard Error. The inter-individual error = 0.492.

PRIMARY outcome

Population: Population PK analysis set included all participants who received at least 1 dose of study drug and have evaluable PK data.

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 first compartment and V2/F is the volume of the second compartment. V2/F of fulvestrant is reported. The measure of variability presented is the inter-individual error.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Mean Volume of Distribution (V2/F) of Fulvestrant	32700 Litres Standard Error NA The measure of variability presented is the inter-individual error, not the Standard Error. The inter-individual error = 0.296.

PRIMARY outcome

Timeframe: Day 1 through 68.7 weeks (maximum observed duration)

Population: The FAS population included participants who received at least 1 dose of study drug.

An adverse event (AE) is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. A serious adverse event (SAE) is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. The TEAEs are defined as events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) Any TEAEs	27 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) Any TESAEs	9 Participants

PRIMARY outcome

Timeframe: Day 1 through 68.7 weeks (maximum observed duration)

Population: The FAS population included participants who received at least 1 dose of study drug.

Number of participants with abnormal clinical laboratory parameters reported as TEAEs are reported. Clinical laboratory parameter analysis included hematology and clinical chemistry.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs Anemia	1 Participants
Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs Vitamin D Deficiency	1 Participants

PRIMARY outcome

Timeframe: Day 1 through Month 12 of treatment period

Population: The FAS population included participants who received at least 1 dose of study drug.

Number of participants with compliance to study treatment are reported. Treatment compliance was ensured at each treatment visit whether each participant received all protocol-defined injections up until the point they either withdrew from the study or completed the main study period. Compliance with study treatment for each participant for the 12-month treatment period was calculated as total number of injections divided by number of visits between first injection (Month 0) and last injection (at Month 11).

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Number of Participants With Compliance to Study Treatment	30 Participants

PRIMARY outcome

Timeframe: Day 1 through 68.7 weeks (maximum observed duration)

Population: The FAS population included participants who received at least 1 dose of study drug.

Number of participants with withdrawals from study treatment due to TEAE are reported.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Number of Participants With Withdrawals From Study Treatment Due to TEAE	0 Participants

PRIMARY outcome

Timeframe: Month 12 (final visit) of treatment period

Population: he FAS population included participants who received at least 1 dose of study drug. Here, number of participants analyzed denotes those participants who were evaluable at the specified time point.

Serum oestradiol level at Month 12 (final visit) is reported.

Outcome measures

Outcome measures
Measure	Fulvestrant n=26 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Hormone Assay: Serum Oestradiol Level	25.95 pmol/L Standard Deviation 30.718

PRIMARY outcome

Timeframe: Month 12 (final visit) of treatment period

Serum LH level collected at Month 12 (final visit) is reported.

Outcome measures

Outcome measures
Measure	Fulvestrant n=28 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Hormone Assay: Serum Luteinizing Hormone (LH) Level	0.11 IU/L Standard Deviation 0.042

PRIMARY outcome

Timeframe: Month 12 (final visit) of treatment period

Serum FSH level collected at Month 12 (final visit) is reported.

Outcome measures

Outcome measures
Measure	Fulvestrant n=29 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Hormone Assay: Serum Follicle-stimulating Hormone (FSH) Level	1.13 IU/L Standard Deviation 1.024

PRIMARY outcome

Timeframe: Month 12 (final visit) of treatment period

Serum testosterone level at Month 12 (final visit) is reported.

Outcome measures

Outcome measures
Measure	Fulvestrant n=28 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Hormone Assay: Serum Testosterone Level	0.65 nmol/L Standard Deviation 0.273

SECONDARY outcome

Timeframe: From Baseline (Month 0) through Month 12 treatment period

Population: The FAS population included participants who received at least 1 dose of study drug.

Change in Tanner stage (measure of pubertal progression) of breast from baseline to Month 12/last visit is reported. Tanner stage (breast) is a score of range 1-5 where 1 = no development and 5 = adult breast.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Tanner Stage of Breast From Baseline to Month 12/Final Visit	0.0 Unit on a scale Interval -3.0 to 2.0

SECONDARY outcome

Timeframe: From Baseline (Month 0) through Month 12 treatment period

Population: The FAS population included participants who received at least 1 dose of study drug.

Change in Tanner stage (measure of pubertal progression) of pubic hair from baseline to Month 12/final visit is reported. Tanner stage (pubic hair) is a score of range 1-5 where 1 = no development and 5 = adult pubic hair.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Tanner Stage of Pubic Hair From Baseline to Month 12/Final Visit	0.0 Unit on a scale Interval -2.0 to 1.0

SECONDARY outcome

Timeframe: From Baseline (screening visit) through Month 12 treatment period

Population: The FAS population included participants who received at least 1 dose of study drug. Here, number of participants analyzed denotes those participants who were analyzed for this endpoint.

Change in PAH for children over age 6 is reported. Bone age radiographs were collected retrospectively. PAH equals the current height divided by a factor (the fraction of final adult height) based on current bone age (central read) and current bone age relative to chronological age, classified as retarded, average or advanced. Retarded is defined as current bone age (years) \< chronological age (years) minus 1; advanced is defined as current bone age (years) \> chronological age (years) plus 1; otherwise, bone age is classified as average. The PAH was summarized using the Bayley and Pinneau method.

Outcome measures

Outcome measures
Measure	Fulvestrant n=17 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Change in Predicted Adult Height (PAH) From Baseline to Month 12/Final Visit	0.5 Centimeter Standard Deviation 4.10

SECONDARY outcome

Timeframe: Baseline (screening)

Population: The FAS population included participants who received at least 1 dose of study drug.

The 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 cyclic adenosine monophosphate (cAMP) formation, which in the gonads, results in episodic uncontrolled sex steroid production and subsequent pubertal development. For participants who provided separate specific informed consent, the percentage of participants with a Gsα mutation at screening was assessed by molecular analysis of peripheral blood.

Outcome measures

Outcome measures
Measure	Fulvestrant n=30 Participants Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
Percentage of Participants With McCune-Albright Syndrome (MAS) Associated G Protein α-subunit (Gsα) Mutation	23.3 Percentage of participants

Adverse Events

Fulvestrant

Serious events: 9 serious events

Other events: 25 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Fulvestrant n=30 participants at risk Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
General disorders Pyrexia	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Bronchitis	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Viral Infection	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Injury, poisoning and procedural complications Femur Fracture	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Metabolism and nutrition disorders Dehydration	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Arthralgia	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Bone Pain	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Pain In Extremity	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Nervous system disorders Dizziness	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Nervous system disorders Neuromyopathy	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Psychiatric disorders Tic	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Reproductive system and breast disorders Ovarian Cyst	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Wheezing	3.3% 1/30 • Number of events 1 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.

Other adverse events

Other adverse events
Measure	Fulvestrant n=30 participants at risk Participants received intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants were dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants were dosed at 4 mg/kg) into the buttock or thigh monthly for 12 months or until the participant demonstrates lack of efficacy based upon one or more of the primary endpoints or experiences a serious drug-related toxicity requiring treatment discontinuation.
General disorders Pyrexia	46.7% 14/30 • Number of events 32 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
General disorders Injection Site Inflammation	13.3% 4/30 • Number of events 4 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
General disorders Fatigue	6.7% 2/30 • Number of events 2 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
General disorders Injection Site Pain	6.7% 2/30 • Number of events 6 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Gastrointestinal disorders Abdominal Pain	26.7% 8/30 • Number of events 21 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Gastrointestinal disorders Vomiting	26.7% 8/30 • Number of events 12 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Gastrointestinal disorders Diarrhoea	16.7% 5/30 • Number of events 7 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Gastrointestinal disorders Abdominal Pain Upper	13.3% 4/30 • Number of events 7 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Gastrointestinal disorders Toothache	10.0% 3/30 • Number of events 6 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Gastrointestinal disorders Nausea	6.7% 2/30 • Number of events 6 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Nervous system disorders Headache	26.7% 8/30 • Number of events 20 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Nervous system disorders Lethargy	6.7% 2/30 • Number of events 2 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Cough	23.3% 7/30 • Number of events 10 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Oropharyngeal Pain	13.3% 4/30 • Number of events 6 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Rhinitis	20.0% 6/30 • Number of events 14 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Bronchitis	6.7% 2/30 • Number of events 2 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Ear Infection	10.0% 3/30 • Number of events 5 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Gastroenteritis	10.0% 3/30 • Number of events 3 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Otitis Media	6.7% 2/30 • Number of events 8 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Pharyngitis	6.7% 2/30 • Number of events 3 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Pharyngitis Streptococcal	6.7% 2/30 • Number of events 3 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Sinusitis	6.7% 2/30 • Number of events 2 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations H1N1 Influenza	6.7% 2/30 • Number of events 4 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Tonsillitis	10.0% 3/30 • Number of events 3 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Upper Respiratory Tract Infection	16.7% 5/30 • Number of events 8 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Vaginal Infection	6.7% 2/30 • Number of events 2 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Varicella	6.7% 2/30 • Number of events 3 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Pain In Extremity	16.7% 5/30 • Number of events 18 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Arthralgia	6.7% 2/30 • Number of events 2 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Bone Pain	6.7% 2/30 • Number of events 3 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Neck Pain	6.7% 2/30 • Number of events 3 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Ear and labyrinth disorders Ear Pain	13.3% 4/30 • Number of events 9 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Urinary Tract Infection	10.0% 3/30 • Number of events 3 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Metabolism and nutrition disorders Decreased Appetite	10.0% 3/30 • Number of events 4 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Skin and subcutaneous tissue disorders Rash	10.0% 3/30 • Number of events 4 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Skin and subcutaneous tissue disorders Eczema	6.7% 2/30 • Number of events 2 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Productive Cough	6.7% 2/30 • Number of events 2 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Vascular disorders Hot Flush	6.7% 2/30 • Number of events 2 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
General disorders Injection Site Reaction	6.7% 2/30 • Number of events 5 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.
Infections and infestations Nasopharyngitis	13.3% 4/30 • Number of events 6 • Day 1 through 68.7 weeks (maximum observed duration) The FAS population included participants who received at least 1 dose of study drug.

Additional Information

Global Clinical Lead

AstraZeneca Clinical study Information Center

Phone: +1-877-240-9479

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee MedImmune has 60 days to review results communications prior to public release and may delete information that compromises ongoing studies or is considered proprietary. This restriction is not intended to compromise the objective scientific integrity of the manuscript, it being understood that results shall be published regardless of outcome.
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