24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

• Ages 2 to 30 years
• All sexes
• No healthy volunteers
• Child, Adult

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

About this study

Official title: VATCH (Vascular Anomaly Analysis for Therapy Choice): Phase II Study of Alpelisib Treatment in Subjects With TIE2/PIK3CA Pathway Driven Vascular Anomalies

Conditions

Vascular Anomalies, Vascular Anomaly, TIE2/PIK3CA Pathway Driven Vascular Anomalies

Being tested

alpelisib (BYL719)

Keywords

vascular anomalies, complex vascular anomalies, CaNVAS, PIK3CA, TIE-2/TEK, PIK3CA inhibitor, alpelisib

Medical subject headings

Vascular Malformations, Hereditary Sensory and Autonomic Neuropathies

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

• alpelisib (BYL719)Drug

  Also called vijoice, BYL719

  Subjects will receive oral alpelisib daily in continuous 28-day cycles. Patients aged 18 years and older will start at 125 mg/day with a maximum dose of 250 mg/day; patients aged 6 - 17 years will start at 50 mg/day with a maximum dose of 200 mg/day. A single dose reduction will be permitted in individual subjects who experience toxicity while still having evidence of clinical benefit and is assessed per the investigator.

What is being measured

The primary outcome is the main question the study is designed to answer.

1. Percentage of subjects with a Substantial Response or Intermediate Response to Alpelisib using an Individualized Response Criteria

   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. "Substantial Response" = Improvement in Radiologic assessment by 20%, AND improvement in Global Health PROs by 3T-score points, AND improvement in at least 1 CBA, AND no clinically meaningful worsening of disease).

   Each subject will be determined to have: 1) Substantial Response, or 2) Intermediate Response, or 3) Stable disease, or 4) Worsening disease. A subject that is found to have a Substantial Response or Intermediate Response is considered to have a "beneficial response." Statistical analyses will be run to determine the percentage of subjects with a beneficial response.

   Measured: 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)

2. Relative size of target lesion(s) as determined by radiologic assessment

   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 by alternative quantitative imaging modality (e.g. X-ray or ultrasound) this may be substituted and used as alternative imaging. Up to 3 lesions will be identified as target lesions and recorded and measured at screening. The same method of assessment and the same technique will be used to characterize each target lesion at the specified follow-up timepoints. CBA can be substituted for radiologic evaluation in subjects without radiologically evaluable disease.

   Measured: Measured at screening and after cycles 6, 12, and 24. Each cycle is 28 days.

3. Quality of life as evaluated by PROMIS Patient Reported Outcome (PRO) questionnaire T-scores

   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. Change in quality of life will be evaluated and applied to the Individualized Response Criteria using the PROMIS global health T-scores.

   Measured: 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.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1. First posted22 Apr 2026
2. Study start27 Aug 2025
3. Primary completion27 Oct 2029 (estimated)
4. Study completion27 Oct 2030 (estimated)

Resources

• Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
• Other trials for Vascular AnomaliesStudies of the same condition, open and under way.
• More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
• What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.