Phase 2
N=30
Faslodex in McCune-Albright Syndrome
Puberty, Precocious · McCune-Albright Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00278915 ↗Enrolled (actual)
30
Serious AEs
30.0%
Results posted
Jul 2011
Primary outcome: Primary: Change in Frequency of Annualized Days of Vaginal Bleeding on Treatment Compared to Baseline — -3.6 Days per year
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Fulvestrant (Drug)
- Age
- Pediatric · 1+ yrs
- Sex
- Female
- Sponsor
- AstraZeneca
- Primary completion
- Dec 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Frequency of Annualized Days of Vaginal Bleeding on Treatment Compared to Baseline |
-3.6 | — |
| PRIMARY 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 | — |
| PRIMARY Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over a 6-month Treatment Period |
78.3 | — |
| PRIMARY Percentage of Participants With Baseline Vaginal Bleeding Who Experienced Cessation of Vaginal Bleeding Over the Whole 12-month Treatment Period |
34.8 | — |
| PRIMARY Change in Rate of Bone Age (BA) Advancement Over First 6-month Treatment Period Compared to Baseline |
-0.83 | — |
| PRIMARY Change in Rate of BA Advancement Over Second 6-month Treatment Period Compared to Baseline |
-1.10 | — |
| PRIMARY Change in Rate of BA Advancement Over the Whole 12-month Treatment Period Compared to Baseline |
-0.93 | — |
| PRIMARY Change in Growth Velocity (Annualized Growth Velocity in cm/Year) Over First 6-month Treatment Period Compared to Baseline |
-1.7 | — |
| PRIMARY Change in Growth Velocity (Annualized Growth Velocity in cm/Year) Over Second 6-month Treatment Period Compared to Baseline |
-0.8 | — |
| PRIMARY Change in Growth Velocity (Annualized Growth Velocity in cm/Year) Over Whole 12-month Treatment Period Compared to Baseline |
-1.4 | — |
| PRIMARY Change in Growth Velocity (Z-score) Over the First 6-month Treatment Period Compared to Baseline |
-1.60 | — |
| PRIMARY Change in Growth Velocity (Z-score) Over the Second 6-month Treatment Period Compared to Baseline |
-0.64 | — |
| PRIMARY Change in Growth Velocity (Z-score) Over the Whole 12-month Treatment Period Compared to Baseline |
-1.14 | — |
| PRIMARY Change in Uterine Volume From Baseline to Month 6 as Assessed by Ultrasound |
-1.10 | — |
| PRIMARY Change in Uterine Volume From Month 6 to Month 12/Final Visit as Assessed by Ultrasound |
-0.13 | — |
| PRIMARY Change in Uterine Volume From Baseline to Month 12/Final Visit as Assessed by Ultrasound |
-2.44 | — |
| PRIMARY Change in Mean Ovarian Volume From Baseline to Month 6 as Assessed by Ultrasound |
0.10 | — |
| PRIMARY Change Mean in Ovarian Volume From Month 6 to Month 12/Final Visit as Assessed by Ultrasound |
0.76 | — |
| PRIMARY Change in Mean Ovarian Volume From Baseline to Month 12/Final Visit as Assessed by Ultrasound |
1.01 | — |
| PRIMARY Mean Clearance of Fulvestrant |
38.4 | — |
| PRIMARY Mean Volume of Distribution (V1/F) of Fulvestrant |
33000 | — |
| PRIMARY Mean Volume of Distribution (V2/F) of Fulvestrant |
32700 | — |
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) |
27; 9 | — |
| PRIMARY Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs |
1; 1 | — |
| PRIMARY Number of Participants With Compliance to Study Treatment |
30 | — |
| PRIMARY Number of Participants With Withdrawals From Study Treatment Due to TEAE |
— | — |
| PRIMARY Hormone Assay: Serum Oestradiol Level |
25.95 | — |
| PRIMARY Hormone Assay: Serum Luteinizing Hormone (LH) Level |
0.11 | — |
| PRIMARY Hormone Assay: Serum Follicle-stimulating Hormone (FSH) Level |
1.13 | — |
| PRIMARY Hormone Assay: Serum Testosterone Level |
0.65 | — |
| SECONDARY Change in Tanner Stage of Breast From Baseline to Month 12/Final Visit |
0.0 | — |
| SECONDARY Change in Tanner Stage of Pubic Hair From Baseline to Month 12/Final Visit |
0.0 | — |
| SECONDARY Change in Predicted Adult Height (PAH) From Baseline to Month 12/Final Visit |
0.5 | — |
| SECONDARY Percentage of Participants With McCune-Albright Syndrome (MAS) Associated G Protein α-subunit (Gsα) Mutation |
23.3 | — |
Summary
The purpose of this study is to evaluate the safety, effectiveness and pharmacokinetics of a study drug called Faslodex (fulvestrant) in the treatment of progressive precocious puberty (PPP) (early puberty) in girls with McCune-Albright syndrome (MAS)
Eligibility Criteria
Inclusion Criteria
- Females less than or equal to 10 years of age (prior to 11th birthday)
- Diagnosis of MAS
- PPP associated with MAS
Exclusion Criteria
- Received any prior treatment for PPP associated with MAS with fulvestrant
- Abnormal platelet count or liver function tests
- Bleeding disorders
- Long term anticoagulation therapy
- Known hypersensitivity to any component of the study drug
Data sourced from ClinicalTrials.gov (NCT00278915). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.