MedTrace Logo

Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum

NCT04589650 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 206

Last updated 2026-04-30

Study results available
· View outcomes & findings →

Summary

This is a prospective Phase II multi-center study with an initial 16-week, randomized, double-blind, placebo-controlled period, followed by two extension periods to assess the efficacy, safety and pharmacokinetics (PK) of alpelisib in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)

Conditions

  • PIK3CA-related Overgrowth Spectrum (PROS)

Interventions

DRUG

Alpelisib

Adult participants (group 1) will receive 125 mg of alpelisib oral tablets once daily. Pediatric participants (Group 2: 6 to 17 years old) will receive 50 mg of alpelisib oral tablets once daily. Pediatric participants (Group 4: 2 to 5 years old) will receive 50 mg of alpelisib oral tablets once daily, Pediatric participants (Group 3: 0 to 5 years old) will receive alpelisib granules formulation with an age-dependent starting dose and maximum dose levels ranging from 20 mg every other day to 50 mg once daily. Pediatric participants (Group 5: 6 to 17 years old) will receive 125 mg of alpelisib oral tablets once daily.

DRUG

Placebo

Participants will receive matching placebo once daily up to week 16.

Sponsors & Collaborators

Principal Investigators

  • Novartis Pharmaceuticals · Novartis Pharmaceuticals

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
0 Days
Max Age
100 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-04-19
Primary Completion
2024-03-20
Completion
2031-01-09
FDA Drug
Yes

Countries

  • United States
  • Canada
  • China
  • France
  • Germany
  • Hong Kong
  • Italy
  • Netherlands
  • Norway
  • Spain
  • Switzerland
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04589650 on ClinicalTrials.gov