Treatment for lymphatic malformations with a specific genetic mutation

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the effectiveness of alpelisib, a targeted therapy, in treating lymphatic malformations associated with a PIK3CA mutation in both pediatric and adult patients. The study is divided into two stages: the first stage focuses on determining the appropriate dosage of alpelisib through a 24-week open-label phase, while the second stage involves a randomized, double-blind, placebo-controlled phase to confirm efficacy and safety. Participants will be monitored for changes in radiological response and symptom severity throughout the trial. The study aims to provide a comprehensive assessment of the treatment's impact on this specific patient population.

Why it matters

Who can join: the full rules

Key inclusion criteria:

1. Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian.
2. 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.
3. 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).
4. 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.
5. Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization.
6. Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization.
7. 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.

Key exclusion criteria:

1. Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent.
2. 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.
3. Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent.
4. Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent.
5. 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

Where it's running

• UCSF Benioff Children s Hospital
  Oakland, California, United States Recruiting
• Childrens Hospital of Orange County
  Orange, California, United States Recruiting
• Lucile Packard Childrens Hosp
  Palo Alto, California, United States Recruiting
• Childrens National Medical Center
  Washington D.C., District of Columbia, United States Recruiting
• Nemours Childrens Clinic
  Jacksonville, Florida, United States Recruiting
• Childrens Hosp Boston Dept of Heme
  Boston, Massachusetts, United States Recruiting

+11 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.