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)

NCT06975618 · tracked via the Priya Life Science China tracker

Sponsor

Haihe Biopharma Co., Ltd.

Phase

Phase 1/2

Started

2023-08-22

Last updated

2026-06-29

Condition(s) studied

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

Investigational drug(s) / intervention(s)

CYH33Placebo

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.

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.

Study 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

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

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.

Primary outcome measure(s)

Phase I: The maximum tolerated dose (MTD) and/or phase II recommended dose (RP2D) — 27 weeks
To evaluate the safety and tolerability of CYH33 and determine the maximum tolerated dose (MTD) and/or phase II recommended dose (RP2D) of CYH33 in adult and adolescent patients
Phase II PROS Cohort: BIRC-assessed objective response rate (ORR) at Week 24 — Baseline to 24weeks
Proportion of patients achieving ≥20% reduction from baseline in the sum of target lesion volumes, with no progression of non-target lesions and no new lesions, as assessed by blinded independent review committee (BIRC).
Phase II PRVM Cohort: BIRC-assessed objective response rate (ORR) at Week 24 — Baseline to 24weeks
Proportion of patients achieving ≥20% reduction from baseline in the sum of target lesion volumes, with no progression of non-target lesions and no new lesions, as assessed by blinded independent review committee (BIRC).

Trial sites (15)

Facility	City	Region	Status
Capital Center for Children's Health, Capital Medical University	Beijing	Beijing Municipality	Recruiting
Plastic Surgery Hospital, Chinese Academy of Medical Sciences	Beijing	Beijing Municipality	Recruiting
Fujian Medical University Union Hospital	Fuzhou	Fujian	Recruiting
Guangzhou Women and Children's Medical Center	Guangzhou	Guangdong	Not Yet Recruiting
Henan Provincial People's Hospital	Zhengzhou	Henan	Recruiting
The Second Xiangya Hospital of Central South University	Changsha	Hunan	Recruiting
Shanghai Ninth People Hospital, Shanghai Jiaotong University School of Medicine	Shanghai	Shanghai Municipality	Recruiting
West China Hospital of Sichuan University	Chengdu	Sichuan	Recruiting
Tonan Hospital	Sapporo	Hokkaido	Recruiting
National Hospital Organization Kobe Medical Center	Kobe	Hyōgo	Recruiting
Yokohama City University Hospital	Yokohama	Kanagawa	Recruiting
Tohoku University Hospital	Sendai	Miyagi	Recruiting
Shinshu University Hospital	Matsumoto	Nagano	Recruiting
Kyorin University Hospital	Mitaka	Tokyo	Recruiting
Gifu University Hospital	Gifu	Japan	Recruiting

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Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06975618 on ClinicalTrials.gov ↗ ← All trials in China