Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT05948943 · ClinicalTrials.gov registry record · Phase 2
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
A Phase 2 study of Lymphatic Malformations, sponsored by Novartis Pharmaceuticals.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 232
- Enrollment target
- 20
- Study locations
NCT05948943: Recruiting Phase 2 study of Lymphatic Malformations, sponsored by Novartis Pharmaceuticals.
NCT05948943 is a Phase 2 study of Lymphatic Malformations that is actively recruiting participants, run by Novartis Pharmaceuticals. The registered enrollment target is 232 participants, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (74% higher). The trial reports 20 study locations across 12 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05948943, a Phase 2 study of Lymphatic Malformations, is actively recruiting participants, sponsored by Novartis Pharmaceuticals.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 232 participants
- Enrollment target
- 20
- Study locations
Study Summary
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.
Primary Outcome
Radiological response defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a BIRC at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions. The percentage of participants with a radiological response at Week 24 of Stage 2 in adult and pediatric (6-17 years of age) groups will be assessed
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Alpelisib
Study Locations (20)
Other
- Novartis Investigative Site - Capital Federal
- Novartis Investigative Site - Brussels
- Novartis Investigative Site - Bordeaux
- Novartis Investigative Site - Bron
Ohio
- Cinn Children Hosp Medical Center - Cincinnati
- Univ Hospital Of Cleveland - Cleveland
- Cleveland Clinic Foundation - Cleveland
California
- UCSF Benioff Children s Hospital - Oakland
- Lucile Packard Childrens Hosp - Palo Alto
Texas
- Baylor College Of Medicine - Houston
- U of TX Health Science Ct - Houston
New South Wales
- Novartis Investigative Site - Sydney
- Novartis Investigative Site - Sydney
District of Columbia
- Childrens National Medical Center - Washington D.C.
Massachusetts
- Childrens Hosp Boston Dept of Heme - Boston
Missouri
- WA Uni School Of Med - St Louis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 232 participants |
| Start Date | 2023-11-24 |
| Est. Completion | 2033-05-02 |
| Phase | Phase 2 |
What NCT05948943 shows while recruiting
NCT05948943 is an interventional study that assigns participants to a tested intervention. The registered 232 participants enrollment target is mid-sized for trials with a published cap, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (74% higher).
The record links to 1 condition, with Lymphatic Malformations appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT05948943 names 20 study sites across 12 states, led by Other, Ohio, California.
Frequently Asked Questions
What is clinical trial NCT05948943 about?
NCT05948943 is a clinical study titled "Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.". 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.
What is the current status of trial NCT05948943?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 232 participants. The study started on 2023-11-24. Estimated completion is 2033-05-02.
What conditions does trial NCT05948943 study?
This clinical trial studies the following conditions: Lymphatic Malformations.
What interventions are being tested in trial NCT05948943?
The interventions under investigation include: Placebo (DRUG), Alpelisib (DRUG).
Who is sponsoring clinical trial NCT05948943?
This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05948943 being conducted?
This trial has 20 study locations across California, District of Columbia, Massachusetts, Missouri, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Lymphatic Malformations
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05948943's enrollment target sits among peer trials
232 256th of 2000 higher than 1,744 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT01356290 · 232 participants · Phase 2
Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors
- NCT04753359 · 232 participants · NA
Mediterranean Diet and Weight Loss: Targeting the Bile Acid/Gut Microbiome Axis to Reduce Colorectal Cancer
- NCT04770870 · 232 participants · NA
Post Approval Study2: Hintermann Series H3 Total Ankle Replacement System
- NCT06213649 · 232 participants · Phase 3
Parasitic Ulcer Treatment Trial
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI