EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)

Conditions

• PIK3CA-related Overgrowth Spectrum (PROS)

Eligibility Criteria

Sex

ALL

Age

2 Years - 100 Years

Healthy Volunteers

Not accepted

Interventions

• Alpelisib — DRUG
  Oral Film-Coated Tablet (FCT): * Group 1: 250 mg once daily * Group 2, 6 to \<18 years: 125 mg once daily (starting dose) Granules: • Group 2, 2 to \<6 years: 50 mg once daily (starting dose)

Study Details

This study is designed to demonstrate the efficacy and assess safety and tolerability of oral daily alpelisib in participants with PIK3CA-related overgrowth spectrum (PROS).

Key Dates

Start date

Oct 9, 2025

Status verified

Jun 2026

Primary completion

Aug 2, 2028

Completion

Sep 30, 2030

Study Design

Enrollment

104 participants (estimated)

Allocation

NA

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Group 1
  Adult participants ≥18 years of age.
• Experimental: Group 2
  Children and adolescents 2 to \<18 years of age.

Primary Outcome Measure

Proportion of participants with a confirmed objective response by BIRC [ Time Frame: Up to Week 48 ]

Central Contacts

• Novartis Pharmaceuticals
  1-888-669-6682
  [email protected]
• Novartis Pharmaceuticals
  +41613241111
  [email protected]

Locations (4)

Find similar trials in St Louis, MO

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