Phase
Condition
Holoprosencephaly
Abnormal Blood Vessels (Arteriovenous Malformations)
Birth Defects
Treatment
Placebo
RLY-2608
Clinical Study ID
Ages > 2 All Genders
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
The participant must have a clinical diagnosis of PROS or a malformation within theISSVA classification.
One or more documented activating PIK3CA mutation(s) that are targeted by selectivePI3Kα inhibitors in lesional tissue and/or cell-free DNA from the lesion or blood.Some participants may be eligible without a documented PIK3CA mutation as long as noother genetic driver has been documented.
Lansky (<16 yo) or Karnofsky (≥16 yo) performance status of ≥50.
Agree to provide archived lesional fluid and/or tissue or be willing to undergopretreatment lesional biopsy (if considered safe and medically feasible) to assessPIK3CA status.
Exclusion
Key Exclusion Criteria:
History of hypersensitivity to PI3K inhibitors.
Any factors that increase the risk of QTc prolongation or risk of arrhythmic events
Clinically significant, uncontrolled cardiovascular disease
Received disease-directed therapy prior to the first dose of study drug:
Systemic therapy or antibody within 5 half-lives of the therapy.
Local therapy including radiation, surgery, or other procedures within 28 days;lesion(s) must have demonstrated progression after the procedure.
Study Design
Connect with a study center
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United StatesActive - Recruiting
Washington University School of Medicine
St. Louis, Missouri 63110
United StatesActive - Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United StatesActive - Recruiting
Texas Children's Hospital
Houston, Texas 77030
United StatesActive - Recruiting
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