ClinicaliQ Trial Snapshot
- Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation. — Recruiting • Phase II • Oncology • NCT05948943.
- What is being tested: Alpelisib (a PI3K inhibitor) administered as film-coated tablets in pediatric and adult patients with lymphatic malformations (LyM) caused by PIK3CA mutations, evaluated against placebo for radiological response and symptom severity.
- Patient eligibility overview: The trial includes both pediatric and adult patients who have lymphatic malformations confirmed to be associated with PIK3CA mutations, representing a genetically-defined patient population with a specific molecular driver of disease.
Use This Page For
- Quick orientation before opening the registry record.
- Checking recruitment status, phase and sponsor at a glance.
- Connecting this trial to nearby guidelines, Drug Science and education.
What This Trial Is Studying
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.
Eligibility Snapshot
- Key inclusion criteria: 1. 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.
Use the source registry for the full inclusion and exclusion criteria before discussing referral or enrolment.