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
NCT ID: NCT06789913
Last Updated: 2026-01-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
277 participants
INTERVENTIONAL
2025-06-13
2031-10-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
Part 2: Part 2 will explore the clinical activity of RLY-2608 at 1 or more adult, adolescent, and pediatric recommended Phase 2 dose (RP2D) in various populations of participants with PROS and malformations associated with PIK3CA mutations in an open-label basket trial design.
Part 3: In Part 3, 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 the Group 1 and 2 RP2Ds versus placebo. Randomization will be stratified based on indication, and prior systemic therapy.
TREATMENT
NONE
Study Groups
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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.
RLY-2608
RLY-2608 is a mutant-selective, oral PI3Kα inhibitor.
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.
RLY-2608
RLY-2608 is a mutant-selective, oral PI3Kα inhibitor.
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.
RLY-2608
RLY-2608 is a mutant-selective, oral PI3Kα inhibitor.
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.
RLY-2608
RLY-2608 is a mutant-selective, oral PI3Kα inhibitor.
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.
RLY-2608
RLY-2608 is a mutant-selective, oral PI3Kα inhibitor.
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.
RLY-2608
RLY-2608 is a mutant-selective, oral PI3Kα inhibitor.
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.
RLY-2608
RLY-2608 is a mutant-selective, oral PI3Kα inhibitor.
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.
Placebo
RLY-2608 matched-placebo
Interventions
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RLY-2608
RLY-2608 is a mutant-selective, oral PI3Kα inhibitor.
Placebo
RLY-2608 matched-placebo
Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
2 Years
ALL
No
Sponsors
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Relay Therapeutics, Inc.
INDUSTRY
Responsible Party
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Locations
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Phoenix Children's Hospital
Phoenix, Arizona, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
University of California, Los Angeles
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
University of California, San Francisco
San Francisco, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Children's Hospital of Atlanta
Atlanta, Georgia, United States
Riley Children's Hospital
Indianapolis, Indiana, United States
Johns Hopkins Medical Institute
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
UNC Chapel Hill
Chapel Hill, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Cleveland Clinic Children's
Cleveland, Ohio, United States
Texas Children's Hospital
Houston, Texas, United States
University of Wisconsin, Madison
Madison, Wisconsin, United States
Sydney Children's Hospital, Randwick
Randwick, New South Wales, Australia
Monash Health
Clayton, Victoria, Australia
Murdoch Children's Research Institute
Parkville, Victoria, Australia
UC Louvain
Ottignies-Louvain-la-Neuve, , Belgium
Ospedale Pediatrico Bambino Gesù IRCCS
Roma, , Italy
Hospital Sant Joan de Deu
Barcelona, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Great Ormond Street Hospital for Children
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Role: primary
Other Identifiers
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2024-518895-30-00
Identifier Type: CTIS
Identifier Source: secondary_id
RLY-2608-201
Identifier Type: -
Identifier Source: org_study_id
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