Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA M… (NCT05948943) | Clinical Trial Compass
RecruitingPhase 2/3
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
United States232 participantsStarted 2023-11-24
Plain-language summary
The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo.
Who can participate
Age range0 Years – 100 Years
SexALL
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Inclusion criteria
✓. Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian.
✓. Participant must be willing to remain at the clinical site as required by the protocol and be willing to adhere to study restrictions and examination schedules.
✓. Participant has a physician confirmed and documented diagnosis of a symptomatic LyM at the time of informed consent (Note: the physician must confirm that the LyM cannot be included under the PROS diagnostic criteria).
✓. Participant is not considered as a candidate for or is not willing to receive non-drug therapies including but not limited to sclerotherapy, embolization, and surgery until the completion of Week 24 in Stage 1 and 2.
✓. Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization.
✓. Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization.
✓. Participants must be able to ingest study drug (either in tablet form or as a drinkable suspension \[Groups 1 to 4\] or granules or as an oral suspension \[Group 5\]) as assessed within 7 days before study treatment start. Drug administration via feeding tubes is allowed.
Exclusion criteria
✕. Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent.
✕. Participant has a physician-confirmed and documented diagnosis of a Central Conducting Lymphatic Anomaly, General Lymphatic Anomaly, Gorham-Stout disease, Kaposiform lymphangiomatosis at the time of informed consent.
What they're measuring
1
Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
✕. Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent.
✕. Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent.
✕. Participant had previous treatment with alpelisib and/or any other PI3K inhibitors with treatment duration longer than 2 weeks at the time of informed consent.