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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)

RecruitingPhase 1, Phase 2Ages any - anyAllHaihe Biopharma Co., Ltd.
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In plain words

What the trial is, and whether it could suit you

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)

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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 โ‰ฅ18 years old (or meet the legal adult age according to local regulations), and adolescent patients should be โ‰ฅ12 years old and \<18 years old (or meet the legal definition of adolescent according to local regulations; additionally, adolescent patients should weigh โ‰ฅ35 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.
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What it tests

  • CYH33
  • Placebo
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Where

15 sites
Capital Center for Children's Health, Capital Medical University
Beijing, China
Plastic Surgery Hospital, Chinese Academy of Medical Sciences
Beijing, China
Fujian Medical University Union Hospital
Fuzhou, China
Guangzhou Women and Children's Medical Center
Guangzhou, China
Henan Provincial People's Hospital
Zhengzhou, China
The Second Xiangya Hospital of Central South University
Changsha, China
Shanghai Ninth People Hospital, Shanghai Jiaotong University School of Medicine
Shanghai, China
West China Hospital of Sichuan University
Chengdu, China

โ€ฆand 7 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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