Evaluating CYH33 for PIK3CA-related overgrowth and vascular malformations
A Phase I/II, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacokinetic Characteristics, and Efficacy of CYH33 (a Selective PI3Kα Inhibitor) in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)
This study is testing a new treatment called CYH33 to see if it can help people with PIK3CA-related overgrowth and vascular problems feel better and have fewer side effects.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 141 (estimated) |
| Sex | All |
| Sponsor | Haihe Biopharma Co., Ltd. Industry-sponsored |
| Locations | 4 sites (Beijing, Beijing Municipality and 3 other locations) |
| Trial ID | NCT06975618 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates include patients aged 12 years and older diagnosed with PROS or PRVM who meet the study's eligibility criteria.
Not a fit: Patients with significant cardiovascular disease or those who have previously received PI3K or AKT inhibitors may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from PIK3CA-related overgrowth and vascular malformations.
How similar studies have performed: While this approach is novel, similar studies targeting PIK3CA-related conditions have shown promise, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Beijing, Beijing Municipality and 3 other locations
- Capital Institute of Pediatrics — Beijing, Beijing Municipality, China (Recruiting)
- Plastic Surgery Hospital, Chinese Academy of Medical Sciences — Beijing, Beijing Municipality, China (Recruiting)
- Shanghai Ninth People Hospital, Shanghai Jiaotong University School of Medicine — Shanghai, Shanghai Municipality, China (Recruiting)
- West China Hospital of Sichuan University — Chengdu, Sichuan, China (Recruiting)
Study contacts
- Study coordinator: Xiaoxi Lin, MD
- Email: linxiaoxi@126.com
- Phone: +86-13701997136
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.