A Phase I/II, multicenter study to evaluate the safety, tolerability, pharmacokinetics characteristics, and efficacy of CYH33 in patinets with PIK3CA related overgrowth spectrum (PROS) and PIK3CA related vascular malformations (PRVM) (A Phase I/II study of CYH33 in Patients with PROS and PRVM)

未提供0 个研究点目标入组 81 人开始时间: 待定

适应症

试验速览

阶段: 不适用
状态: 招募中
入组人数: 81

研究概览研究设计入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional
分配方式: Randomized

入排标准

年龄范围: 12age old over 至 No limit（—）
性别: All

入选标准

•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 vascular malformation and/or overgrowth syndrome (PRVOs), including 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 (For the Phase II Study PROS Cohort, patients with PIK3CA mutation confirmed by local laboratory test may proceed to clinical screening and can be enrolled if eligible, but must simultaneously provide fresh or archived tissue samples for retrospective confirmation of PIK3CA mutation by central laboratory test, whereas for the Phase II Study PRVM Cohort, patients with PIK3CA mutation confirmed by local laboratory test may proceed to clinical screening but must provide fresh or archived tissue samples, and enrollment is permitted only after central laboratory confirmation of PIK3CA mutation), with at least one measurable lesion related to PROS or PRVM. For the Phase II Study, measurable lesions must be confirmed by central imaging assessment before patients can be enrolled
•a)The diagnosis of PROS should meet the clinical diagnostic criteria, such as Klippel-Trenaunay syndrome (KTS), CLOVES syndrome, CLAPO syndrome, diffuse capillary malformation with overgrowth (DCMO), megalencephaly-capillary malformation (MCAP), hemihyperplasia-multiple lipomatosis (HHML), fibroadipose vascular anomaly (FAVA), facial infiltrating lipomatosis (FIL), fibroadipose hyperplasia or overgrowth (FAO), or other PROS subtypes, and should be determined by the investigator to require systemic treatment;
•b) PRVM should meet one of the following clinical diagnostic types, simple vascular malformations, such as lymphatic malformation (LM), venous malformation (VM), combined vascular malformations, such as capillary-venous malformation (CVM), capillary-lymphatic malformation (CLM), lymphatic-venous malformation (LVM), capillary-lymphatic-venous malformation (CLVM), or other vascular malformations, and should be determined by the investigator to require systemic treatment.
•The criteria for requiring systemic treatment as determined by the investigator are as follows (both conditions must be met),
•Clinical symptoms: Accompanied by complaints, clinical symptoms or functional limitations affecting the patient's daily life (e.g., pain, fatigue, inability to walk normally, etc.)
•Treatment options, The patient is unsuitable or unwilling to undergo conventional treatments such as surgery, sclerotherapy, or interventional therapy, or has experienced treatment failure/relapse after receiving these therapies.

排除标准

•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.
•Patients who have received any systemic treatment for PROS or PRVM (including surgery, sclerotherapy, laser therapy, interventional therapy, local injection therapy, etc.) within 8 weeks prior to the first dose of study drug, or any drug treatment for PROS or PRVM (e.g., mTOR inhibitors, AKT inhibitors) within 28 days prior to the first dose of study drug, or who have not recovered from the serious side effects.
•Patients who have previously received any PI3K inhibitor (e.g., alpelisib) treatment. If the efficacy of previous PI3K inhibitor treatment was inadequate for reasons other than disease progression, eligibility for enrollment may be determined after evaluation by the investigator.
•Clinically significant PROS/PRVM-related thrombotic events (>=Grade 2, according to NCI-CTCAE v5.0) occurring within 8 weeks prior to signing the informed consent form, and/or sclerotherapy/embolization for vascular complications within 8 weeks prior to signing the informed consent form. Patients receiving anticoagulants for the treatment of PROS/PRVM related coagulation disorders and primary or secondary prevention of thrombosis may be enrolled in the study.
•Patients who have received blood transfusion or platelet infusion therapy within 2 weeks prior to study drug administration. Patients who have been receiving erythropoietin or darbepoetin treatment at least 2 weeks prior to enrollment may be included and allowed to continue such treatment.

研究者

发起方

未提供

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