A Phase II Double-blind Multi-center, Placebo-controlled Trial, to Assess the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP) (SESAM)

NCT05577754 Phase 2 RECRUITING

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)

Details

Lead sponsor	Centre Hospitalier Universitaire Dijon
Phase	Phase 2
Status	RECRUITING
Enrolment	20
Start date	2022-11-28
Completion	2027-03

Conditions

• Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)

Interventions

• Alpelisib (BYL719)
• Matching placebo
• Optional lumbar puncture + blood sample

Primary outcomes

• Proportion of participants with response at end of treatment assessed by the Vineland II Adaptive Behavior Scale (VABS-II) — At 24 months of treatment compared to baseline
  Response defined by an improvement of at least 4 points in the Vineland II Adaptive Behavior Scale (VABS-II). The VABS-II consists of a form which will be filled during an interview with an adult who is familiar with the everyday activities of the patient (usually the parents). It is organized within a three-domain structure: communication, daily living skills, and socialization. In addition, VABS-II has a motor skills domain for the youngest children (younger than 6) and an optional maladaptive behavior index \[Sparrow et al.,2016\]. The domains (communication, daily living skills, and socialization) - standard scores have a mean of 100 and a standard deviation of 15. Adaptive levels can also be determined. A global standard score can also be computed (the Adaptive Behavior Composite standard score) and also has a mean of 100 and a standard deviation of 15.

Countries

France