Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With … (NCT05577754) | Clinical Trial Compass
RecruitingPhase 2
Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP)
France20 participantsStarted 2022-11-28
Plain-language 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)
Who can participate
Age range2 Years – 40 Years
SexALL
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Inclusion criteria
✓. Signed informed consent and assent (when applicable) from the patient, parent, or guardian must be obtained prior to any study related screening procedures are performed.
✓. Male or female patients age ≥2 years and ≤40 years at the time of informed consent
✓. Patients with diagnosis of MCAP\* with neurodevelopmental disorder presentation (from specific learning disorder to severe intellectual disability)
✓. Documented evidence of a postzygotic or constitutional mutation(s) in the PIK3CA gene performed in local laboratories using a Deoxyribonucleic acid (DNA) based validated test at the time of informed consent.
✓. Adequate bone marrow and organ function (assessed during the screening visit):
✓. Absolute neutrophil count ≥ 1.5 × 109/L
✓. Platelets ≥ 100 × 109/L
✓. Hemoglobin ≥ 9.0 g/dL (transfusions are allowed)
Exclusion criteria
✕. Patient previously treated with alpelisib
✕. Known impairment of GI function due to concomitant disease that may significantly alter the absorption of the study drug (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection) at time of informed consent.
✕. Participant with uncontrolled diabetes mellitus (Type I or II) at time of informed consent.
What they're measuring
1
Proportion of participants with response at end of treatment assessed by the Vineland II Adaptive Behavior Scale (VABS-II)
Timeframe: At 24 months of treatment compared to baseline
✕. History of hypersensitivity to any drugs or metabolites of PI3K inhibitor or any of the excipients of alpelisib at time of informed consent.
✕. Participant with other concurrent severe and/or uncontrolled medical conditions that would, in the treating Physician's judgment, contraindicate administration of alpelisib (e.g., active and/or uncontrolled severe infection, chronic active hepatitis, hepatic impairment Child Pugh score C, immuno-compromised, etc.) at time of informed consent.
✕. Female participants of childbearing potential and male participants who do not agree at time of informed consent to abstinence or, if sexually active, unwilling to use a condom and/or a highly effective method of contraception for the duration of the study and for one week following discontinuation of alpelisib. Highly effective contraception methods is one of the following:
✕. Total abstinence: when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
✕. Female sterilization: have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or bilateral tubal ligation at least six weeks before taking alpelisib. In case of oophorectomy alone, only when the reproductive status of the female has been confirmed by follow-up hormone level assessment