Evaluating CYH33 for PIK3CA-related overgrowth and vascular malformations

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a multi-center, open-label, single-arm study designed to assess the safety, tolerability, pharmacokinetics, and preliminary efficacy of CYH33 in patients diagnosed with PIK3CA-related overgrowth spectrum (PROS) and PIK3CA-related vascular malformations (PRVM). The study will enroll both adult and adolescent patients, who will be monitored for their response to the treatment and any potential side effects. The aim is to gather data that could inform future treatment options for these conditions.

Why it matters

Who can join: the full rules

Key inclusion criteria:

1. The patient or the patient's legal guardian (if applicable) voluntarily signs the Informed Consent Form.
2. At the time of signing the informed consent, adult patients should be ≥18 years old (or meet the legal adult age according to local regulations), and adolescent patients should be ≥12 years old and \<18 years old (or meet the legal definition of adolescent according to local regulations; additionally, adolescent patients should weigh ≥35 kg).
3. The patient is diagnosed with PIK3CA-related overgrowth spectrum (PROS) or PIK3CA-related vascular malformations (PRVM), and provides a report confirming PIK3CA mutation detected by local laboratory or the Sponsor-designated central laboratory, with at least one measurable lesion related to PROS or PRVM.
4. Patients should demonstrate adequate organ and bone marrow function during the 28-day screening period.

Key exclusion criteria:

1. PROS patients presenting solely with isolated macrodactyly, epidermal nevi/nevus, and megalencephaly (only one clinical feature or any combination of these three features) without other PROS-related lesions.
2. Patients who have received any systemic treatment for PROS or PRVM within 8 weeks prior to the first dose of study drug, or any drug treatment for PROS or PRVM (e.g., mTOR inhibitors) within 28 days prior to the first dose of study drug.
3. Patients who have previously received any PI3K inhibitor treatment.

Where it's running

• Capital Center for Children's Health, Capital Medical University
  Beijing, Beijing Municipality, China Recruiting
• Plastic Surgery Hospital, Chinese Academy of Medical Sciences
  Beijing, Beijing Municipality, China Recruiting
• Fujian Medical University Union Hospital
  Fuzhou, Fujian, China Recruiting
• Henan Provincial People's Hospital
  Zhengzhou, Henan, China Recruiting
• The Second Xiangya Hospital of Central South University
  Changsha, Hunan, China Recruiting
• Shanghai Ninth People Hospital, Shanghai Jiaotong University School of Medicine
  Shanghai, Shanghai Municipality, China Recruiting

Study contacts

• Study coordinator: Xiaoxi Lin, MD
• Email: linxiaoxi@126.com
• Phone: +86-13701997136

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Alpelisib for children and adults with PIK3CA-related overgrowth spectrum (PROS)

  This project will try daily oral alpelisib to see if it reduces overgrowth and improves symptoms in children and adults with PROS.

  Recruiting at 41 sites Phase 2 Novartis · Sacramento, California and 40 other locations · NCT06997588

• Treatment for PIK3CA-related overgrowth and malformations using RLY-2608

  This study is testing a new treatment called RLY-2608 to see if it can help adults and children with PIK3CA-related overgrowth and malformations feel better.

  Recruiting at 40 sites Phase 2 Relay Therapeutics, Inc. · Phoenix, Arizona and 39 other locations · NCT06789913

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.