NCT07543822 Alpelisib Vascular Malformations Clinical Landscape Report 2026: Design, Endpoints, Sponsor and Readout Outlook

Turn a newly registered trial into a decision-ready clinical landscape. This report examines NCT07543822 using PatSnap Clinical Trials MCP for protocol design and endpoint evidence, with Drug & Asset MCP and Company & Deal Intelligence MCP used as the companion asset and sponsor enrichment workflow. Explore PatSnap MCP Servers to reproduce the research sequence inside an AI workflow.

MCP evidence snapshot: 13 August 2026. Trial records, recruitment status and projected dates can change after the snapshot and should be rechecked before operational decisions.

Why NCT07543822 is a hot trial to watch

Vascular Malformations is being segmented by mechanism, treatment setting, geography and endpoint architecture. NCT07543822 is notable because it evaluates Alpelisib in a Phase 2 design sponsored by The Children's Hospital of Philadelphia. The main development question is whether the protocol can translate its rationale into a clinically interpretable and operationally credible readout.

Trial landscape snapshot

Protocol design and endpoint interpretation

The study will enroll participants 2 months of age up to 30 years of age. The purpose of this study is to assess the effectiveness and safety of Alpelisib (the "Study Drug") in patients with PIK3CA/TIE-2/TEK pathway driven vascular anomalies (VA). Alpelisib has been approved by the U.S. Food and Drug Administration (FDA) for treating adults and children with certain types of breast cancer. Its use in this study is considered experimental because FDA has not approved the study drug for treating people with VAs. Study participation will last for up to 3 years and will involve regular study visits to Children's Hospital of Philadelphia (CHOP)'s Philadelphia Campus. Participants will need to take the study drug Alpelisib for at least 2 years, or up to 3 years in total if there is a positive response. Participating in this research means you will attend up to 16 clinic visits for the purposes of the study. Most visits will take approximately

Allocation is N/A, masking is None (Open Label), and the intervention model is Sequential Assignment. Planned enrollment of [object Object] participants across United States shapes statistical precision, execution risk and external validity. Interpretation should account for baseline risk, prior therapy, assessment schedule, missing-data handling and clinical relevance—not only statistical significance.

• Percentage of subjects with a Substantial Response or Intermediate Response to Alpelisib using an Individualized Response Criteria (Individualized Response Criteria will be evaluated after 6 cycles (each cycle is 28 days) and then every 6 months until the end of the study (approximately 1.5 years)) — Individualized Response is a composite outcome determined by the combined results of the following 3 distinct study assessments after cycle 6: Radiologic evaluation, PROMIS Patient reported outcome (PRO) measurements, and Clinical Benefit Assessments (CBA). The results of these 3 distinct assessments will be evaluated using a set of specific criteria to determine the Individualized Response to treatment (i.e. "Substa
• Relative size of target lesion(s) as determined by radiologic assessment (Measured at screening and after cycles 6, 12, and 24. Each cycle is 28 days.) — \*\*For subjects with radiologically evaluable disease only\*\* The target lesion(s) will be measured at screening using imaging modality of choice based on underlying vascular anomaly (e.g. Magnetic Resonance Imaging (MRI), Magnetic Resonance Lymphangiography (MRL), US, X-rays, CT scan). MRI or MRL imaging will be recommended as primary imaging modality. For subjects with disease manifestation better characterized b
• Quality of life as evaluated by PROMIS Patient Reported Outcome (PRO) questionnaire T-scores (Begin at screening and after cycles 6, 12, and 24. If subject enters extension phase, will continue to measure every 6 cycles until end of therapy (approximately 1 year). Each cycle is 28 days.) — Patient Reported Outcomes Measurement Information System (PROMIS) questionnaires will be distributed to subjects and caregivers (if applicable.) A raw score will be calculated for each PROMIS subscale. Raw scores will then be translated into a T-score metric with mean of 50 (standard deviation of 10). A higher T-score means more of the outcome being measured. Global health measures will be used as the primary outcome

Readout outlook and evidence gap

The current protocol points to [object Object] as the best available readout proxy. Because this is an active or newly posted study, a trial-specific result citation is not included until a normalized clinical-trial-result record becomes available. That gap is itself operationally important: teams should monitor first result indexing, conference abstracts, protocol amendments and changes to the primary-completion date.

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Asset and sponsor context

Drug & Asset MCP profile: Alpelisib is indexed as No normalized modality returned, with target No normalized target returned, mechanism No normalized mechanism returned, and global highest development status No normalized global status returned.

Company & Deal Intelligence MCP profile: The Children's Hospital of Philadelphia is resolved to a normalized organization record in PHILADELPHIA COUNTY, United States. The organization workflow adds identity, location, portfolio and partnering context where available.

The sponsor profile matters because scientific rationale alone does not determine development value. Manufacturing readiness, portfolio fit, geographic reach, partnering capacity and the ability to fund confirmatory development can determine whether a positive signal becomes a competitive asset.

White space around this program

• Sharper patient selection: prospective biomarker or phenotype definitions that identify who is most likely to benefit.
• Clinically interpretable endpoints: outcomes connecting activity with function, symptoms, survival or treatment burden.
• Comparator relevance: evidence against the most decision-relevant contemporary standard of care.
• Broader external validity: evidence across additional geographies, demographic groups and real-world settings.
• Operational differentiation: a development path that closes the readout gap without sacrificing safety or durability.

What to monitor next

Monitor recruitment, enrollment changes, protocol amendments, endpoint hierarchy, primary-completion timing, first result indexing, asset ownership and sponsor partnerships. A change in endpoint, population or ownership can alter the probability of success before a headline data release.

Bottom line

NCT07543822 provides a focused lens on Vascular Malformations development. Its value will be determined by whether Alpelisib can convert the current Phase 2 design into evidence that is clinically meaningful, operationally credible and differentiated from competing programs.

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