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)

Haihe Biopharma

Status and phase: Enrolling; Phase 2; Phase 1

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

Treatments: Drug: CYH33

Study type: Interventional

Funder types: Industry

Identifiers

NCT06975618

CYH33-G208

Details and patient eligibility

About

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)

Enrollment: 130 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• The patient or the patient's guardian (if applicable) voluntarily signs the Informed Consent Form.
• At the time of signing the Informed Consent, the age of adult patients should be ≥ 18 years, and the age of adolescent patients should be ≥ 12 years and < 18 years of age.
• Patient diagnosed with PIK3CA-related overgrowth spectrum (PROS) or PIK3CA-related vascular malformation (PRVM).
• Patients should be tested to have adequate organ and bone marrow function, within 28 days of the screening period.

Exclusion criteria

• Patients who have received any prior treatment with PI3K or AKT inhibitors.
• Received a blood transfusion or platelet transfusion within 2 weeks prior to the administration of the investigational drug.
• Patients with clinically significant cardiovascular disease.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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130 participants in 1 patient group

Cohort1：Adult cohort:； Cohort2：Adolescent cohort；

Experimental group

Description:

Cohort1：Adult cohort: Participants will receive oral CYH33 once daily. An initial dose of 10 mg QD and possible additional dose levels (e.g. 15mg, 20mg QD) will be evaluated in the dose escalation stage and dose expansion stage in Phase I. For Phase II, the starting dose of CYH33 will be selected based on the RP2D determined in the Phase I study. Cohort2：Adolescent cohort: Participants will receive oral CYH33 once daily. An initial dose of 5 mg QD and possible additional dose levels (e.g. 10mg, 15mg QD) will be evaluated in the dose escalation stage and dose expansion stage in Phase I. For Phase II, the starting dose of CYH33 will be selected based on the RP2D determined in the Phase I study.

Treatment:

Drug: CYH33