Actively Recruiting
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
Led by Relay Therapeutics, Inc. · Updated on 2026-05-05
277
Participants Needed
31
Research Sites
13 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of RLY-2608, a mutant-selective PI3Kα inhibitor, in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations caused by PIK3CA mutations. This Phase 2 study has three parts: a dose selection phase, an exploratory phase with various participant groups, and a randomized, double-blind study comparing RLY-2608 to placebo. The study includes multiple subpopulations to understand how the drug works across ages and conditions related to PIK3CA mutations. Participants receive oral doses of RLY-2608 tailored for their age group. Children aged 2 to under 12 undergo dose escalation to find the appropriate dose, while those 12 years and older receive fixed doses. The study has single-arm dose expansion cohorts for different age groups, followed by a randomized phase where participants aged 6 and older receive either RLY-2608 or placebo. Dosing schedules and group assignments are carefully designed to assess safety and response in various participant groups. Throughout the study, participants undergo regular assessments including lesion volume measurements, safety labs, ECGs, and evaluations of side effects. Researchers collect samples to confirm PIK3CA mutation status and monitor plasma drug levels. Patient-reported outcomes and quality of life questionnaires are also used. The main outcomes include identifying the recommended dose, tracking adverse events, and measuring changes in lesion size and symptoms over time. Participation continues with regular monitoring until study completion or discontinuation, which may last several months or longer.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Clinical diagnosis of PROS or a malformation within the ISSVA classification
- One or more documented activating PIK3CA mutation(s) in lesional tissue or cell-free DNA, or eligibility without documented PIK3CA mutation if no other genetic driver is identified
- Lansky performance status below 16 years old or Karnofsky performance status 16 years and older of 50 or higher
- Willingness to provide archived lesional fluid and/or tissue or undergo pretreatment lesional biopsy if safe and feasible
You will not qualify if you...
- History of hypersensitivity to PI3K inhibitors
- Factors increasing risk of QTc prolongation or arrhythmic events
- Clinically significant, uncontrolled cardiovascular disease
- Received disease-directed systemic therapy or antibody within 5 half-lives before first study drug dose
- Received local therapy including radiation, surgery, or other procedures within 28 days before first study drug dose unless lesion shows progression
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants receive oral doses of the study drug RLY-2608 or placebo as assigned in a randomized design.
Visits at the start and end of each treatment cycle, approximately every 4 weeks
Duration - Up to study completion
Participants are monitored for treatment response and safety with assessments approximately every 3 months for the first year, then every 6 months during treatment.
Approximately quarterly visits initially, then biannual visits
Trial Site Locations
Total: 31 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
3
University of California, Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
4
Stanford University
Palo Alto, California, United States, 94304
Actively Recruiting
5
University of California, San Francisco
San Francisco, California, United States, 94158
Actively Recruiting
6
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
7
Children's Hospital of Atlanta
Atlanta, Georgia, United States, 30329
Actively Recruiting
8
Riley Children's Hospital
Indianapolis, Indiana, United States, 46202
Actively Recruiting
9
Johns Hopkins Medical Institute
Baltimore, Maryland, United States, 21287
Actively Recruiting
10
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
11
Corewell Health
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
12
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
13
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
14
UNC Chapel Hill
Chapel Hill, North Carolina, United States, 27599
Actively Recruiting
15
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
16
Cleveland Clinic Children's
Cleveland, Ohio, United States, 44195
Actively Recruiting
17
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
18
Seattle Children's Hospital
Seattle, Washington, United States, 98101
Actively Recruiting
19
University of Wisconsin, Madison
Madison, Wisconsin, United States, 53715
Actively Recruiting
20
Sydney Children's Hospital, Randwick
Randwick, New South Wales, Australia, 2031
Actively Recruiting
21
Children's Health Queensland Hospital and Health
South Brisbane, Queensland, Australia, 4101
Actively Recruiting
22
Monash Health
Clayton, Victoria, Australia, 3168
Actively Recruiting
23
Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
Actively Recruiting
24
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
Actively Recruiting
25
UC Louvain
Ottignies-Louvain-la-Neuve, Belgium
Actively Recruiting
26
University of Halle
Halle, Germany, Germany
Actively Recruiting
27
Ospedale Pediatrico Bambino Gesù IRCCS
Roma, Italy
Actively Recruiting
28
A.O.U Città della Salute e della Scienza di Torino
Torino, Italy
Actively Recruiting
29
Hospital Sant Joan de Deu
Barcelona, Spain
Actively Recruiting
30
Hospital Universitario La Paz
Madrid, Spain
Actively Recruiting
31
Great Ormond Street Hospital for Children
London, United Kingdom
Actively Recruiting
Research Team
R
Relay Therapeutics, Inc
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
8
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