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Study Assessing Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study CBYL719F12002 (EPIK-P1)

NCT04980833 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 41

Last updated 2026-04-02

No results posted yet for this study

Summary

This is a prospective interventional Phase II multi center study, open label, preceded by a retrospective non-interventional period, to assess the long-term safety and efficacy of alpelisib, in pediatric and adult participants with PROS.

Conditions

  • PIK3CA-related Overgrowth Spectrum (PROS)

Interventions

DRUG

Alpelisib

Participant's treatment plan has been established by the treating physician, within the global compassionate use framework. Doses permitted are 50, 125, 200 and 250 mg.

Sponsors & Collaborators

Principal Investigators

  • Novartis Pharmaceuticals · Novartis Pharmaceuticals

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
100 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-01-27
Primary Completion
2027-08-20
Completion
2027-08-20
FDA Drug
Yes

Countries

  • United States
  • France
  • Ireland
  • Spain

Study Locations

More Related Trials

Entities

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 NCT04980833 on ClinicalTrials.gov