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)

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

141

Participants

Timeline

Start Date

August 22, 2023

Primary Completion Date

December 31, 2029

Study Completion Date

December 31, 2029

Conditions
PIK3CA-Related Overgrowth Spectrum (PROS)PIK3CA-related Vascular Malformations (PRVM)
Interventions
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.

Trial Locations (4)

100000

RECRUITING

Capital Institute of Pediatrics, Beijing

100144

RECRUITING

Plastic Surgery Hospital, Chinese Academy of Medical Sciences, Beijing

200011

RECRUITING

Shanghai Ninth People Hospital, Shanghai Jiaotong University School of Medicine, Shanghai

610041

RECRUITING

West China Hospital of Sichuan University, Chengdu

Sponsors
All Listed Sponsors
lead

Haihe Biopharma Co., Ltd.

INDUSTRY