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Clinical Trials in Japan / NCT06975618
Recruiting Phase 1/2

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 Japan 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)

CYH33 →Placebo

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
No
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)

FacilityCityRegionStatus
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

More Haihe Biopharma Co., Ltd. trials in Japan

A Study to Evaluate the Efficacy and Safety of CYH33 in Patients With Recurrent/Persistent Ovary Clear Cell Carcinoma Active, not recruiting
Assessment of Anti-tumor and Safety in Glumetinib in Patients With c-MET-positive Non-Small Cell Lung Cancer 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 Japan