A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation

Launched by RELAY THERAPEUTICS, INC. · Jan 17, 2025

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ClinConnect Summary

This clinical trial is studying a new medication called RLY-2608, which is designed to help people with a condition called PIK3CA-Related Overgrowth Spectrum (PROS) and other related malformations caused by a specific genetic mutation known as PIK3CA. The trial aims to find out if RLY-2608 is effective and safe for adults and children who have these conditions. Participants will be randomly assigned to receive either the medication or a placebo (a dummy treatment that looks the same but has no active ingredients) in a double-blind manner, meaning neither the participants nor the researchers will know who is receiving which treatment.

To be eligible for the trial, participants need to have a clinical diagnosis of PROS or a related malformation, and they must have a confirmed PIK3CA mutation. They should also have a performance status score of 50 or higher, which indicates they are able to carry out some daily activities. Participants will need to provide samples from their lesions or agree to a biopsy to help assess their condition. The trial is currently not recruiting participants but will be open to individuals of all ages. It's important to note that certain health conditions, such as significant heart problems or allergies to similar medications, may exclude someone from participating. If you or a loved one is considering joining this trial, it could be a potential opportunity to receive a new treatment for a challenging condition.

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Eligibility criteria

• 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 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:
• • 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:
• • 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.