Ph2 Study of RLY-2608 in PROS and PIK3CA Driven Malformations
Research type
Research Study
Full 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
IRAS ID
1011468
Contact name
Carmen Shin
Contact email
Sponsor organisation
Relay Therapeutics, Inc.
Clinicaltrials.gov Identifier
Research summary
The phosphatidylinositol-4,5-bisphosphate-3 kinase, catalytic subunit alpha gene (PIK3CA) mutations can occur early in life, causing a progressive group of disorders, called PIK3CA related overgrowth spectrum (PROS) and other related malformations. These mutations activate an enzyme that can cause overgrowth or malformations in a patient’s body. There are currently no curative treatments available globally for patients with these conditions. RLY-2608 is an investigational medication designed to inhibit the activity of the enzyme leading to those disorders. RLY-2608 will be tested in this study to see if it can help adult, adolescent, and pediatric patients with PROS and malformations caused by PIK3CA mutations.
Study RLY-2608-201 consists of:
Part 1: Open-label dose selection,
Part 2: Open-label basket design, and
Part 3: Double-blind study comparing the efficacy and safety of RLY-2608 versus placebo
In Part 1, enrollment will begin with adults and adolescents but may be extended to younger age groups in a staggered manner based on experience with the older patients. During Part 2, patients will receive recommended dose(s) based on Part 1. During Part 3, patients will receive RLY-2608 (or placebo) at the recommended dose(s) based on findings from Part 1 and Part 2. The amount of time that patients will stay in the study will depend on how they react and respond to the medication.
Adult, adolescent, and pediatric patients with PROS and other malformations driven by PIK3CA mutation will take part in this study. To be included in this study, patients must meet all study-specific entry criteria.
Overall, available data and experience with RLY-2608 suggest a favorable benefit-risk profile for the treatment of patients with PROS and malformations driven by PIK3CA mutation who otherwise have limited treatment options. The protocol for RLY-2608-201 has been designed to minimize risks to patients through appropriate safety monitoring procedures and assessments.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
25/EM/0095
Date of REC Opinion
17 Jun 2025
REC opinion
Further Information Favourable Opinion