Treatment for PIK3CA-related overgrowth and malformations using RLY-2608

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 2 study evaluates the safety and efficacy of RLY-2608, a mutant-selective PI3Kα inhibitor, in treating adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and related malformations. The study consists of three parts: a dose selection phase, a basket design with exploratory cohorts for various subpopulations, and a randomized, double-blinded comparison against a placebo. Participants will be assessed for their response to the treatment based on their specific PIK3CA mutations. The study aims to provide targeted therapy for individuals affected by these conditions.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

* The participant must have a clinical diagnosis of PROS or a malformation within the ISSVA classification.
* One or more documented activating PIK3CA mutation(s) that are targeted by selective PI3Kα inhibitors in lesional tissue and/or cell-free DNA from the lesion or blood. Some participants may be eligible without a documented PIK3CA mutation, with the sponsor's approval, as long as no other 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 undergo pretreatment lesional biopsy (if considered safe and medically feasible) to assess PIK3CA status.

Key Exclusion Criteria:

* Known hypersensitivity to RLY-2608.
* 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:

  1. Systemic therapy or antibody within 5 half-lives of the therapy.
  2. Local therapy including radiation, surgery, or other procedures within 28 days; lesion(s) must have demonstrated progression after the procedure.

Where it's running

• Phoenix Children's Hospital
  Phoenix, Arizona, United States Recruiting
• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Recruiting
• University of California, Los Angeles
  Los Angeles, California, United States Recruiting
• Stanford University
  Palo Alto, California, United States Recruiting
• University of California, San Francisco
  San Francisco, California, United States Recruiting
• Children's Hospital Colorado
  Aurora, Colorado, United States Recruiting

Study contacts

• Study coordinator: Relay Therapeutics, Inc
• Email: ClinicalTrials@relaytx.com
• Phone: 617-322-0731

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.