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Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP)

NCT05577754 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2026-02-24

No results posted yet for this study

Summary

This study is a two periods multi-center Phase II trial, with a 6 months double-blind, placebo-controlled period followed by open label period, to assess the efficacy and safety of alpelisib (BYL719) in pediatric and adult patients with Megalencephaly-CApillary malformation Polymicrogyria syndrome (MCAP)

Conditions

  • Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)

Interventions

DRUG

Alpelisib (BYL719)

Administration during open label and double-blind period of group B: alpelisib will be taken once a day each day over 24 mois During open label period of group A: alpelisib will be taken once a day each day over 24 months

DRUG

Matching placebo

During double-blind period of group A: matching placebo will be taken once a day each day over 6 months

PROCEDURE

Optional lumbar puncture + blood sample

Between 6 and 24 months of treatment with Alpelisib

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

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

Timeline & Regulatory

Start
2022-11-28
Primary Completion
2027-03-31
Completion
2027-03-31

Countries

  • France

Study Locations

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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 NCT05577754 on ClinicalTrials.gov