What is alpelisib studying for lymphatic malformations?

Alpelisib is being studied to see how it affects lymphatic malformations caused by a PIK3CA mutation. Researchers want to understand its impact on radiological changes and symptom severity. The study is sponsored by Novartis Pharmaceuticals and involves both children and adults.

Who may be eligible for this lymphatic malformations clinical trial?

People of all ages from newborns to 100 years old with a confirmed diagnosis of symptomatic lymphatic malformations and a PIK3CA mutation may be eligible. Participants should not have had previous extended treatment with similar drugs and must be willing to avoid certain surgeries during the study. Please review eligibility criteria for more details.

Is there any cost to participate in this lymphatic malformations clinical trial?

Study-related care, including the study drug or placebo and all trial visits, is provided at no cost. However, personal travel expenses can vary and may not be covered. Participants should plan accordingly for any travel or accommodation costs.

Actively Recruiting

Phase 2

Phase 3

Age: 0Years - 100Years

All Genders

ID05948943

Study of Alpelisib Treatment in Children and Adults With Lymphatic Malformations Caused by PIK3CA Mutation A Two-Stage, Randomized, Placebo-Controlled Trial to Evaluate Effectiveness and Safety

Led by Novartis Pharmaceuticals · Updated on 2026-07-14

232

Participants Needed

Research Sites

256 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the effects of alpelisib film-coated tablets compared to placebo in participants with lymphatic malformations LyM caused by a PIK3CA mutation. This phase IIIII study aims to assess changes in radiological response and symptom severity during treatment. The study includes both pediatric and adult participants and is conducted in multiple centers with a two-stage design. The study has two main stages. Stage 1 is a 24-week open-label phase where adult 18 years and older and pediatric 6 to 17 years participants receive different doses of alpelisib to determine the best doses for Stage 2. Stage 2 is a 24-week randomized, double-blind, placebo-controlled phase to confirm the efficacy and safety of the selected doses in both adults and children aged 6 to 17, followed by an open-label extension. Additionally, Stage 2 includes an open-label core phase for pediatric participants aged 0 to 5 years receiving alpelisib. Participants undergo screening to confirm eligibility, including confirmation of a PIK3CA mutation and measurable lesions. Throughout the study, participants will have radiological assessments, symptom evaluations using patient global impression scales, and monitoring of safety and pharmacokinetics. The study includes regular reviews by an independent committee to ensure safety. The total study duration includes a 24-week core treatment phase and potential extensions for long-term monitoring, lasting up to several years.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 0Years - 100Years

All Genders

Eligibility Criteria

You may qualify if you...

Signed informed consent and assent when applicable
Willingness to remain at the clinical site and follow study restrictions and schedules
Physician-confirmed diagnosis of symptomatic lymphatic malformations not included under PROS criteria
Not a candidate for or unwilling to receive non-drug therapies (sclerotherapy, embolization, surgery) until after Week 24
Evidence of somatic mutation(s) in the PIK3CA gene prior to randomization
At least one measurable lymphatic malformation lesion confirmed by BIRC
Ability to ingest study drug as tablet, suspension, granules, or via feeding tube as assessed before treatment start

You will not qualify if you...

Physician-confirmed diagnosis of PROS at the time of informed consent
Diagnosis of Central Conducting Lymphatic Anomaly, General Lymphatic Anomaly, Gorham-Stout disease, or Kaposiform lymphangiomatosis
History of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis
Established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus
Previous treatment with alpelisib or other PI3K inhibitors for longer than 2 weeks
Other exclusion criteria may apply

Research Team

Novartis Pharmaceuticals

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