Atossa Therapeutics, Inc.
The goal of this clinical trial is to evaluate the safety, tolerability, and blood levels of (Z)-endoxifen in girls with McCune-Albright Syndrome that have peripheral precocious puberty. The main question it aims to answer \[is/are\]: Are there any safety concerns? Is there any evidence of efficacy? There is no comparison group. Participants will: * take a capsule once a day for 3 months; * have doctor visits (tele-health and in-person); * blood samples collected; and * complete questionnaires
Inclusion Criteria: 1. Female child aged ≥ 4 to \< 10 years at the time of informed consent. 2. Body weight ≥ 16 kg. 3. Clinical or molecular diagnosis of McCune-Albright syndrome (MAS), with peripheral precocious puberty and at least one additional characteristic manifestation of MAS other than café-au-lait skin macules, such as polyostotic fibrous dysplasia or another autonomous endocrine manifestation distinct from the participants PPP. 4. Clinical evidence of gonadotropin-independent (peripheral) precocious puberty, including onset of vaginal bleeding before 8 years of age and recurrent v…
(Z)-endoxifen will be administered once daily via the oral route
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