Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.

• Status: Recruiting
• Trial end date: 2030-01-03
• Target enrollment: 0
• Participant gender: All

Summary

The main purpose of this study in participants with PIK3CA-mutated lymphatic malformations (LyM) is to assess the change in radiological response and symptom severity upon treatment with alpelisib as compared to placebo.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals

Criteria

Key Inclusion Criteria:

• 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 LyM at the time of informed consent

• Participant is not considered as a candidate for or is not willing to receive local therapy options including but not limited to sclerotherapy, embolization, and surgery until the completion of Week 24 at the time of informed consent.

• Participant has evidence of a somatic mutation(s) in the PIK3CA gene

• Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization.

Key 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.

• 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.

Other inclusion/exclusion criteria may apply