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OncoMatch/Clinical Trials/NCT05948943

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

Is NCT05948943 recruiting? Yes, currently enrolling (Jun 2026). This Phase 2/3 trial studies Alpelisib for lymphatic malformations.

Phase 2/3RecruitingNovartis PharmaceuticalsNCT05948943Data as of Jun 2026Location: International · 8 countries

Treatment: AlpelisibThe 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.

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Extracted eligibility criteria

Treatments studied

Targeted therapy

Alpelisib

Biomarker criteria

Required: PIK3CA somatic mutation

evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization

Demographics

Ages 0–100

Prior therapy

Cannot have received: PI3K inhibitor (alpelisib)

Exception: treatment duration longer than 2 weeks

previous treatment with alpelisib and/or any other PI3K inhibitors with treatment duration longer than 2 weeks

Structured fields extracted by AI. May contain errors — verify against the official protocol.

US trial sites

  • UCSF Benioff Children s Hospital · Oakland, California
  • Lucile Packard Childrens Hosp · Palo Alto, California
  • Childrens National Medical Center · Washington D.C., District of Columbia
  • Childrens Hosp Boston Dept of Heme · Boston, Massachusetts
  • WA Uni School Of Med · St Louis, Missouri

Showing up to 5 US sites.

See all sites on ClinicalTrials.gov →

Frequently asked questions

Is NCT05948943 currently recruiting?

Yes, this trial is currently recruiting patients.

Are there prior therapy exclusions?

Yes. Prior PI3K inhibitor disqualifies patients from enrollment.

Does this trial require PIK3CA?

Yes, PIK3CA somatic mutation is a required biomarker for enrollment.

Is there an age limit?

Yes. Patients must be 100 years or younger and at least 0 years old.

Could you qualify for this trial?

Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.

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