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.

Recruitment & Eligibility

Status: ot Recruiting

Sex: Female

Target Recruitment: 30

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:
oAn increase of at least one in the breast Tanner Stage over the observation period

and/or
oThe development or persistence of vaginal bleeding during the observation period.
6.At least one of the following two criteria must also be fulfilled:
oAdvanced bone age: defined as, bone age of at least 12 months beyond chronological age at the time of screening.
oRapid growth rate: a growth rate over the observation period that is more than 2 standard deviations above the mean for age, where the growth rate is defined as the change in height (or length) (in cm) divided by the change in time (annualized in years).
7.Patients are eligible provided they are in one of the following categories:
oReceived no previous treatment and have documented retrospective data of at least 6 months for bone age, Tanner Stage, height, weight, and vaginal bleeding (number of bleeding days).
oReceived no previous treatment and do not have documented retrospective data of at least 6 months for bone age, Tanner Stage, height, weight and vaginal bleeding (number of bleeding days) and can be observed for 6 months without treatment (i.e., 6-month observation period).
oDocumented progression on treatment with anastrozole, tamoxifen, testolactone, or other aromatase inhibitors, an anti-androgen, or a progestin requiring immediate treatment provided there exists retrospective data of at least 6 months for bone age, Tanner Stage, height, weight, and vaginal bleeding (number of bleeding days) and are off these agents for 1 month prior to first dose of study drug.
oPreviously treated with any drug for PPP in which therapy was stopped for at least 6 months with subsequent clinical evidence of progression of disease meeting entry criteria No. 5 and 6 and retrospective data of at least 6 months for bone age, Tanner Stage, height, weight, and vaginal bleeding (number of bleeding days).
8.If central precocious puberty (CPP) exists, the patient must have been on a GnRH analog (e.g., Lupron) for at least 6 months prior to study enrollment (date of written consent of parent/legal guardian and patient assent).

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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