An Open-Label, Single-Arm, Phase I/II Platform Trial of Innovative Therapies and Technologies Primarily in Rare Tumors in China (PLATFORM2) in Patients With Advanced Solid Tumors
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 600
- 主要终点
- Objective Response Rate (ORR)
研究概览
简要总结
The goal of this Phase I/II observational and interventional platform study is to evaluate the safety and efficacy of multiple types of innovative anti-tumor drugs and new technologies in patients with rare solid tumors. The study utilizes multi-dimensional precision screening (including WES, RNAseq, mIHC, and quantitative proteomics) to match patients with specific sub-protocols.
Key questions it aims to answer:
Assess the safety of innovative therapies in rare tumor populations. Evaluate the objective response rate (ORR) and other efficacy metrics. Explore biomarkers related to therapeutic efficacy. Participants: Patients with metastatic or advanced rare solid tumors who have failed standard therapy or have no standard treatment options.
详细描述
This is an open-label, non-randomized, multi-arm, single-center Phase I/II platform study (PLATFORM2). Based on the definition of rare tumors in China (incidence < 2.5/100,000 or specific list), eligible patients will undergo multi-omics screening. Based on the molecular profiling results (gene variations or protein expression), patients will be assigned to corresponding treatment arms (Sub-studies). Therapies include small molecules, protein drugs, Cell and Gene Therapy (CGT) products, and therapeutic vaccines. The study employs a Clopper-Pearson Two-Stage Minimax design for each arm.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
BL-B01D1 Arm
Participants receive BL-B01D1 injection according to the protocol-specified dose and schedule until disease progression, unacceptable toxicity, or withdrawal. Safety, efficacy, and biomarker analyses will be conducted throughout the study.
干预措施: BL-B01D1 (Drug)
TAEST1901 Arm
Participants receive a single or fractionated intravenous infusion of TAEST1901 cells according to their assigned dose level, followed by subcutaneous administration of low-dose IL-2. The study primarily evaluates safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy.
干预措施: TAEST1901 (Biological)
VSV Arm
Participants in this arm will receive VSV monotherapy administered according to the protocol-specified dose and schedule. Treatment will continue until radiographic disease progression, unacceptable toxicity, withdrawal of consent, or investigator decision. Tumor response, safety, and exploratory biomarker endpoints will be assessed throughout the study.
干预措施: VSV injection (Drug)
CVL006 Arm
Participants in this arm will receive CVL006 monotherapy administered according to the protocol-specified dose and schedule. Treatment will continue until radiographic disease progression, unacceptable toxicity, withdrawal of consent, or investigator decision. Tumor response, safety, and exploratory biomarker endpoints will be assessed throughout the study.
干预措施: CVL006 (Drug)
IDOV-SAFE Arm
Participants in this arm will receive IDOV-SAFE monotherapy administered according to the protocol-specified dose and schedule. Treatment will continue until radiographic disease progression, unacceptable toxicity, withdrawal of consent, or investigator decision. Tumor response, safety, and exploratory biomarker endpoints will be assessed throughout the study.
干预措施: IDOV-SAFE (Biological)
YL-201 Arm
Participants in this arm will receive YL-201 administered according to the protocol-specified dose and schedule. Treatment will continue until radiographic disease progression, unacceptable toxicity, withdrawal of consent, or investigator decision. Tumor response, safety, and exploratory biomarker endpoints will be assessed throughout the study.
干预措施: YL-201 (Drug)
KXV01 Arm
Participants receive a single intravenous infusion of KXV01 injection according to the protocol-specified dose level until disease progression, unacceptable toxicity, or withdrawal. Safety, efficacy, pharmacokinetic, and biomarker analyses will be conducted throughout the study.
干预措施: KXV01 TCR Lentinvivo Injection (Biological)
MT027 Arm
Participants receive MT027 cell injection via intrapleural injection according to the protocol-specified dose and schedule until the investigator determines no further benefit, unacceptable toxicity, withdrawal of consent, disease progression, death, or loss to follow-up. Safety, tolerability, pharmacokinetics, and preliminary efficacy will be evaluated throughout the study.
干预措施: MT027 (Biological)
QH101 Arm
Participants receive QH101 cell injection via intrathecal infusion or Ommaya reservoir according to the protocol-specified dose and schedule until disease progression, unacceptable toxicity, or withdrawal. Safety, efficacy, and biomarker analyses will be conducted throughout the study.
干预措施: QH101 (Biological)
Meta10-TIL Arm
Participants receive a single intravenous infusion of Meta10-TIL, following non-myeloablative lymphodepletion pretreatment, and subsequent IL-2 administration. Treatment continues until disease progression, unacceptable toxicity, or withdrawal. Safety, efficacy, pharmacokinetic, and pharmacodynamic analyses will be conducted throughout the study.
干预措施: Meta10-TIL (Biological)
TC-N201 Arm
Participants receive TC-N201 injection, a genetically engineered TCR-T cell product targeting NY-ESO-1 and secreting anti-PD-1 single-chain antibody, according to the protocol-specified dose and schedule. Treatment is administered following lymphodepletion chemotherapy and accompanied by interleukin-2 (IL-2) support. Safety, efficacy, pharmacokinetics, and biomarker analyses will be conducted throughout the study.
干预措施: TC-N201 (Biological)
NK510 Arm
Participants receive NK510 injection, a base-edited allogeneic natural killer (NK) cell product with TIGIT knockout, according to the protocol-specified dose and schedule. The study includes both single-dose and multiple-dose phases. Safety, efficacy, pharmacokinetics, immunogenicity, and biomarker analyses will be conducted throughout the study.
干预措施: NK510 (Biological)
CE120 Intratumoral Injection Arm
Participants receive CE120 intratumoral injection according to the protocol-specified dose and schedule (every 2 weeks for a total of 4 doses) until disease progression, unacceptable toxicity, or withdrawal. Safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy will be evaluated throughout the study.
干预措施: CE120 (Drug)
GV20-0251 Arm
Participants receive GV20-0251 injection according to the protocol-specified dose and schedule until disease progression, unacceptable toxicity, or withdrawal. Safety, efficacy, and biomarker analyses will be conducted throughout the study.
干预措施: GV20-0251 (Drug)
YSCH-01 Arm
Participants receive YSCH-01 injection according to the protocol-specified dose and schedule until disease progression, unacceptable toxicity, or withdrawal. Safety, efficacy, and biomarker analyses will be conducted throughout the study.
干预措施: YSCH-01 (Drug)
LYC001 Arm
Participants will receive LYC001 according to the protocol-defined dose and schedule and will continue treatment until disease progression, unacceptable toxicity, or withdrawal. Safety assessments, efficacy evaluations, pharmacokinetic analyses, and biomarker studies will be conducted throughout the study.
干预措施: LYC001 (Drug)
BMD006 Arm
Participants will receive BMD006 according to the protocol-defined dose and schedule and will continue treatment until disease progression, unacceptable toxicity, or withdrawal. Safety assessments and efficacy evaluations will be conducted throughout the study.
干预措施: BMD006 (Drug)
CREPT-618 Arm
Participants will receive CREPT-618 according to the protocol-defined dose and schedule and will continue treatment until disease progression, unacceptable toxicity, or withdrawal. Safety assessments, efficacy evaluations and biomarker studies will be conducted throughout the study.
干预措施: CREPT-618 (Drug)
PRG2505 Arm
Participants will receive PRG2505 according to the protocol-defined dose and schedule and will continue treatment until disease progression, unacceptable toxicity, or withdrawal. Safety assessments, efficacy evaluations , and biomarker studies will be conducted throughout the study.
干预措施: PRG2505 (Biological)
IBI363 Arm
Participants will receive IBI363 according to the protocol-defined dose and schedule and will continue treatment until disease progression, unacceptable toxicity, or withdrawal. Safety assessments, efficacy evaluations , and biomarker studies will be conducted throughout the study.
干预措施: IBI363 (Drug)
结局指标
主要结局
Objective Response Rate (ORR)
时间窗: Up to approximately 2 years.
Assessed by BICR and Investigator per RECIST 1.1.
次要结局
- Progression-Free Survival (PFS)(Up to approximately 2 years.)
- Duration of Response (DoR)(Up to approximately 2 years.)
- Disease Control Rate (DCR)(Up to approximately 2 years.)
- Overall Survival (OS)(Up to 1 year post-treatment.)
- Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)(From enrollment up to 30 days after last dose.)