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
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
This is a 3-part Phase 2 randomized study evaluating the safety and efficacy of the mutant-selective PI3Kα inhibitor, RLY-2608, in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations driven by PIK3CA mutation. Part 1 is a dose selection, Part 2 is a basket design with exploratory single-arm cohorts for various subpopulations of participants, and Part 3 is randomized, double-blinded study vs placebo.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Healthy Volunteers: f
View:
• The participant must have a clinical diagnosis of PROS or a malformation within the ISSVA 2018 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
• 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.
Locations
United States
Arkansas
Arkansas Children's Hospital
RECRUITING
Little Rock
Contact Information
Primary
Relay Therapeutics, Inc
ClinicalTrials@relaytx.com
617-322-0731
Time Frame
Start Date:2025-04
Estimated Completion Date:2031-10
Participants
Target number of participants:280
Treatments
Experimental: Part 1, Group 1
RLY-2608 for patients ≥12 years old with PROS or malformations with PIK3CA mutation. Multiple doses of RLY-2608 for oral administration.
Experimental: Part 1, Group 2
RLY-2608 for participants 6 to \<12 years old with PROS or malformations with PIK3CA mutation.~RLY-2608 will be studied in pediatric participants in a dose escalation design.
Experimental: Part 1, Group 3
Part 1, Group 3: RLY-2608 for participants 2 to \<6 years old with PROS or malformations with PIK3CA mutation.~RLY-2608 will be studied in pediatric participants in a dose escalation design.
Experimental: Part 2, Group 1
Dose expansion single-arm cohorts for various subpopulations of participants ≥12 years old with PROS or malformations with PIK3CA mutation.~Oral dose of RLY-2608 as determined during Part 1.
Experimental: Part 2, Group 2
Dose expansion cohorts for participants 6 to \<12 years old with PROS or malformations with PIK3CA mutation.~Oral dose of RLY-2608 as determined during Part 1.
Experimental: Part 2, Group 3
Dose expansion cohorts for participants 2 to \<6 years old with PROS or malformations with PIK3CA mutation.~Oral dose of RLY-2608 as determined during Part 1.
Experimental: Part 3, Arm 1
Adult (\>18 yo), and adolescent and pediatric (6 to \<18 yo) participants with PROS and malformations with PIK3CA mutation will be randomized to receive RLY-2608 at oral dose determined during Part 1/2 versus placebo.
Placebo_comparator: Part 3, Arm 2
Adult (\>18 yo), and adolescent and pediatric (6 to \<18 yo) participants with PROS and malformations with PIK3CA mutation will be randomized to receive placebo.