Find PIK3CA-Related Overgrowth Spectrum Clinical Trials Near You
A Phase I/II, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacokinetic Characteristics, and Efficacy of CYH33 (a Selective PI3Kα Inhibitor) in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)
Status: Recruiting
Location: See all (4) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Healthy Volunteers: f
View:
• The patient or the patient's legal guardian (if applicable) voluntarily signs the Informed Consent Form.
• 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).
• 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.
• Patients should demonstrate adequate organ and bone marrow function during the 28-day screening period.
Locations
Other Locations
China
Capital Institute of Pediatrics
RECRUITING
Beijing
Plastic Surgery Hospital, Chinese Academy of Medical Sciences
RECRUITING
Beijing
West China Hospital of Sichuan University
RECRUITING
Chengdu
Shanghai Ninth People Hospital, Shanghai Jiaotong University School of Medicine
RECRUITING
Shanghai
Contact Information
Primary
Xiaoxi Lin, MD
linxiaoxi@126.com
+86-13701997136
Time Frame
Start Date:2023-08-22
Estimated Completion Date:2029-12-31
Participants
Target number of participants:141
Treatments
Experimental: Arm 1：Adult cohort:；
Phase I Adult Cohort: Adult patients with PIK3CA-related overgrowth spectrum (PROS) or PIK3CA-related vascular malformations (PRVM) will receive escalating oral doses of CYH33 to determine the maximum tolerated dose (MTD) and/or recommended Phase II dose (RP2D). Expansion cohorts may be opened to further assess safety, tolerability, pharmacokinetics, and preliminary efficacy.
Experimental: Arm 2: Phase I Adolescent Cohort
Phase I Adolescent Cohort: Adolescent patients with PIK3CA-related overgrowth spectrum (PROS) or PIK3CA-related vascular malformations (PRVM) will receive escalating oral doses of CYH33 to determine the maximum tolerated dose (MTD) and/or recommended Phase II dose (RP2D). Expansion cohorts may be opened to further assess safety, tolerability, pharmacokinetics, and preliminary efficacy.
Experimental: Arm 3 : Phase II PROS Cohort
Phase II PROS Cohort: An open-label, single-arm cohort. Adult and adolescent patients with PROS will receive CYH33 at RP2D. Dose escalation may be allowed based on tolerability and clinical assessment. Treatment will continue until disease progression, unacceptable toxicity, or withdrawal.
Experimental: Arm 4 : Phase II PRVM Cohort
Phase II PRVM Cohort: Adult and adolescent patients with PRVM will be randomized 2:1 to CYH33 or placebo during double-blind period. After 8 weeks of treatment, all patients will enter an open-label extension phase to receive CYH33. Treatment continues until disease progression, unacceptable toxicity, or withdrawal.