Trials / Recruiting
RecruitingNCT06975618
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)
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)
- Status
- Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 141 (estimated)
- Sponsor
- Haihe Biopharma Co., Ltd. · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
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)
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | CYH33 | CYH33: Participants will receive oral CYH33 once daily. The starting dose for adults in Phase I is 10 mg QD; adolescents begin at 5 mg QD. In Phase II, patients will receive RP2D determined in the Phase I study. |
| DRUG | Placebo | Placebo: Matching placebo tablets will be administered once daily during the double-blind period of the Phase II PRVM cohort. Patients randomized to placebo will switch to CYH33 at the end of the blinded phase. |
Timeline
- Start date
- 2023-08-22
- Primary completion
- 2029-12-31
- Completion
- 2029-12-31
- First posted
- 2025-05-16
- Last updated
- 2025-09-24
Locations
4 sites across 1 country: China
Source: ClinicalTrials.gov record NCT06975618. Inclusion in this directory is not an endorsement.