EUCTR2005-004893-26-GB
Active, not recruiting
Phase 1
An open-label, non-comparative trial to evaluate the safety, efficacy and pharmacokinetics of FASLODEX (fulvestrant) in girls with progressive precocious puberty associated with McCune-Albright Syndrome.
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Progressive precocious puberty associated with McCune-Albright Syndrome
- Sponsor
- AstraZeneca AB
- Enrollment
- 30
- Status
- Active, not recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\.Provision of written informed consent of parent/legal guardian and subject assent (as required by local law)
- •2\.For patients consenting to participate in the genetic portion of the study, provision of an additional written informed consent specific for DNA sampling and genetic analysis.
- •3\.Females less than or equal to 10 years of age (prior to 11th birthday) at the start of trial therapy (Visit 0\).
- •4\.Diagnosis of McCune\-Albright Syndrome based on having the following clinical criteria:
- •·Precocious puberty evident before the age of 8 years
- •And at least one of the following clinical criteria:
- •·Café au lait spots
- •·Fibrous dysplasia
- •·Presence of Gsa mutation
- •5\.Progressive precocious puberty associated with MAS. Progressive precocious puberty will be defined as:
Exclusion Criteria
- •1\.Male gender
- •2\.Any prior treatment of PPP associated with MAS with fulvestrant
- •3\.Concomitant treatment of PPP associated with MAS, with the exception of bisphosphonates for fibrous dysplasia and GnRH analogs in the case of CPP
- •4\.Liver function tests (AST, ALT) at screening ³ 3´ the upper limit of the reference range for age
- •5\.Platelet count at screening less than 100 ´ 109/L
- •6\.International normalized ratio (INR) greater than 1\.6\.
- •7\.History of bleeding diathesis or long\-term anticoagulant therapy (other than antiplatelet therapy)
- •8\.Any severe concomitant condition that makes it undesirable for the patient to participate in this study
- •9\.Known hypersensitivity to any component of the study drug product
Outcomes
Primary Outcomes
Not specified
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