Trial intelligence / Profile preview

Alpelisib in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)

NCT06997588Phase 2

Key facts

Status: Recruiting
Start: Oct 2025
Primary completion: Jan 2029
Countries: 11
Sponsor: Novartis

Drugs: alpelisib
Conditions: PIK3CA-related overgrowth spectrum (PROS)

Overview

Phase 2, open-label trial of alpelisib in PIK3CA-related overgrowth spectrum (PROS) (2 arms), sponsored by Novartis. Primary endpoint: Objective Response Rate at 48 weeks. Started Oct 2025; status: recruiting.

Design

2 arms

Study design: Non-randomizedOpen-label

Group 1
Adult participants >=18 years of age receiving alpelisib 250 mg once daily.
Group 2
Children and adolescents 2 to <18 years of age receiving alpelisib 50 mg or 125 mg once daily.

Who it enrols

Patients aged >=2 years with symptomatic and progressive PROS, documented PIK3CA somatic mutation, Karnofsky/Lansky >=50, and at least 1 measurable lesion >=2 cm. Excludes patients with only isolated macrodactyly/epidermal nevus/macroencephaly, prior PI3K inhibitor treatment, recent major surgery, or recent radiation.

Endpoints

Primary

Objective Response Rate48 weeks

Secondary

Objective Response Rate in a protocol-defined subset48 weeks
Change from baseline in target lesion volume168 weeks
Change from baseline in MRI-measurable non-target lesion volume168 weeks
Change from baseline in all MRI-measurable lesion volume168 weeks
Appearance of new lesions168 weeks
Proportion of participants with a radiological response168 weeks
Duration of Response3 years
Alpelisib plasma concentration12 weeks

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