NCT06975618 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CYH33 in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)
| NCT ID | NCT06975618 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Haihe Biopharma Co., Ltd. |
| Condition | PIK3CA-Related Overgrowth Spectrum (PROS) |
| Study Type | INTERVENTIONAL |
| Enrollment | 141 participants |
| Start Date | 2023-08-22 |
| Primary Completion | 2029-12-31 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 141 participants in total. It began in 2023-08-22 with a primary completion date of 2029-12-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This study is a multi-center, open-label, single arm, phase I/II study to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of CYH33 in patients with PIK3CA-related overgrowth spectrum (PROS) and PIK3CA-related vascular malformations (PRVM)
Eligibility 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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06975618 clinical trial?
This trial is open to participants of all sexes, studying PIK3CA-Related Overgrowth Spectrum (PROS). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06975618 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06975618 currently recruiting?
Yes, NCT06975618 is actively recruiting participants. Contact the research team at linxiaoxi@126.com for enrollment information.
Where is the NCT06975618 trial being conducted?
This trial is being conducted at Beijing, China, Beijing, China, Fuzhou, China, Guangzhou, China and 11 additional locations.
Who is sponsoring the NCT06975618 clinical trial?
NCT06975618 is sponsored by Haihe Biopharma Co., Ltd.. The trial plans to enroll 141 participants.