Actively Recruiting
A Phase I/II Multicenter Study Evaluating Safety, Pharmacokinetics, and Efficacy of CYH33 for PIK3CA-related Overgrowth Spectrum and Vascular Malformations
Led by Haihe Biopharma Co., Ltd. · Updated on 2025-09-24
141
Participants Needed
4
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, pharmacokinetics, and early effectiveness of CYH33, an oral selective PI3Kα inhibitor, in patients with PIK3CA-related overgrowth spectrum (PROS) and PIK3CA-related vascular malformations (PRVM). This multi-center, open-label, single-arm study includes both adult and adolescent patients and aims to find appropriate dosing and assess treatment responses in these conditions caused by PIK3CA mutations. The study has several cohorts: Phase I adult and adolescent groups receive escalating doses of CYH33 to determine the maximum tolerated dose or recommended Phase II dose. Phase II includes a PROS cohort treated openly with CYH33 at the recommended dose, and a PRVM cohort randomized to CYH33 or placebo for 8 weeks, followed by all patients receiving CYH33 in an open-label extension. Dosing begins at 10 mg daily for adults and 5 mg daily for adolescents, with adjustments allowed based on tolerance and clinical response. Treatment continues until disease progression, unacceptable side effects, or withdrawal. Participants will undergo safety and pharmacokinetic monitoring including drug concentration measurements at multiple time points during the study. Efficacy will be assessed by measuring objective response rates and lesion volumes at defined intervals up to 48 months. Patient-reported outcomes on pain and quality of life will be collected through diaries. The study also tracks adverse events throughout participation. Total involvement varies depending on treatment duration and response, with follow-up assessments scheduled regularly to monitor effects and safety.
CONDITIONS
Brief Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient or legal guardian voluntarily signs informed consent.
- Adult patients aged 18 years or older, or adolescents aged 12 to under 18 years weighing at least 35 kg.
- Diagnosed with PIK3CA-related overgrowth spectrum (PROS) or PIK3CA-related vascular malformations (PRVM) with confirmed PIK3CA mutation and at least one measurable lesion.
- Adequate organ and bone marrow function during the 28-day screening period.
You will not qualify if you...
- PROS patients with only isolated macrodactyly, epidermal nevi/nevus, or megalencephaly without other PROS lesions.
- Patients who received systemic treatment for PROS or PRVM within 8 weeks before the first study drug dose.
- Patients who received any drug treatment for PROS or PRVM, such as mTOR inhibitors, within 28 days prior to the first study drug dose.
- Patients who have previously received any PI3K inhibitor treatment.
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Participants undergo screening assessments to confirm eligibility.
Duration - Up to approximately 48 months or until disease progression, unacceptable toxicity, or withdrawal
Participants receive oral CYH33 once daily to assess safety, tolerability, pharmacokinetics, and preliminary efficacy. In the PRVM cohort, some participants will receive placebo during a double-blind period before switching to CYH33.
Participants have regular visits during dosing and assessments, including baseline and follow-up visits; the double-blind period for the PRVM cohort lasts 8 weeks
Trial Site Locations
Total: 4 locations
1
Capital Institute of Pediatrics
Beijing, Beijing Municipality, China, 100000
Actively Recruiting
2
Plastic Surgery Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, China, 100144
Actively Recruiting
3
Shanghai Ninth People Hospital, Shanghai Jiaotong University School of Medicine
Shanghai, Shanghai Municipality, China, 200011
Actively Recruiting
4
West China Hospital of Sichuan University
Chengdu, Sichuan, China, 610041
Actively Recruiting
Research Team
X
Xiaoxi Lin, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
4
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