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Faslodex in McCune-Albright Syndrome

NCT ID: NCT00278915

Last Updated: 2024-03-05

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-01-31

Study Completion Date

2023-07-20

Brief Summary

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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)

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Detailed Description

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Conditions

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Puberty, Precocious McCune-Albright Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Fulvestrant

Participants will receive intramuscular injection of fulvestrant 2 mg/kg or 4 mg/kg (First 10 participants will be dosed at 2 mg/kg then increased to 4 mg/kg. All subsequent participants will be 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.

Group Type EXPERIMENTAL

Fulvestrant

Intervention Type DRUG

Participants will receive intramuscular injection of fulvestrant as stated in arm description.

Interventions

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Fulvestrant

Participants will receive intramuscular injection of fulvestrant as stated in arm description.

Intervention Type DRUG

Other Intervention Names

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Faslodex ZD9238

Eligibility Criteria

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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
Minimum Eligible Age

1 Year

Maximum Eligible Age

10 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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AstraZeneca

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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AstraZeneca Faslodex Medical Science Director, MD

Role: STUDY_DIRECTOR

AstraZeneca

Locations

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Research Site

Birmingham, Alabama, United States

Site Status

Research Site

Miami, Florida, United States

Site Status

Research Site

Lexington, Kentucky, United States

Site Status

Research Site

Baton Rouge, Louisiana, United States

Site Status

Research Site

The Bronx, New York, United States

Site Status

Research Site

Philadelphia, Pennsylvania, United States

Site Status

Research Site

Salt Lake City, Utah, United States

Site Status

Research Site

Bordeaux, , France

Site Status

Research Site

Bron, , France

Site Status

Research Site

Paris, , France

Site Status

Research Site

Erlangen, , Germany

Site Status

Research Site

Osnabrück, , Germany

Site Status

Research Site

Torino, , Italy

Site Status

Research Site

Moscow, , Russia

Site Status

Research Site

Liverpool, , United Kingdom

Site Status

Research Site

London, , United Kingdom

Site Status

Countries

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United States France Germany Italy Russia United Kingdom

References

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Sims EK, Garnett S, Guzman F, Paris F, Sultan C, Eugster EA; Fulvestrant McCune-Albright study group. Fulvestrant treatment of precocious puberty in girls with McCune-Albright syndrome. Int J Pediatr Endocrinol. 2012 Sep 22;2012(1):26. doi: 10.1186/1687-9856-2012-26.

Reference Type DERIVED
PMID: 22999294 (View on PubMed)

Related Links

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http://filehosting.pharmacm.com/DownloadService.ashx?client=CTR_MED_6111&studyid=404&filename=CSR-D6992C00044.pdf

CSR-D6992C00044.pdf

https://filehosting-v2.pharmacm.com/api/Attachment/Download?tenantId=80217111&amp;parentIdentifier=D6992C00044&amp;attachmentIdentifier=c7f1f38d-0976-4974-9fa9-50e2fcb1b0a4&amp;fileName=Clinical_Study_Report_(CSR)_Synopsis_Redacted.pdf&amp;versionIdentifier=

CSR Synopsis Redacted

Other Identifiers

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2005-004893-26

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

D6992C00044

Identifier Type: -

Identifier Source: org_study_id

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