Clinical Research Directory

A Study of Ultra-fraction Radiotherapy Bridging CART in R/R DLBCL

This is a single-arm single center study to prospectively evaluate the safety and efficacy of ultra-fraction radiotherapy bridging CAR-T therapy in relapsed/refractory diffuse large b cell lymphoma

Long-term Follow-up of Subjects Treated With CAR T Cells

This is a single site, non-randomized, open-label, long-term safety and efficacy follow-up study for Phase 1 studies that evaluate the safety and efficacy of CAR T cells: NCT05660369 (DF/HCC# 22-175) and NCT06026319 (DF/HCC# 23-474).

Clinical Study of CLL-1 CAR-T in the Treatment of Children With R/R AML

A study to evaluate the safety and preliminary efficacy of CLL-1-targeted CAR-T cell therapy in children aged 3 to 18 years with relapsed or refractory acute myeloid leukemia (r/r AML).

Multi-omics Profiling of Patients With Aplastic Anemia Before and After CD7-CAR-T Therapy

Aplastic anemia (AA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and hypocellular bone marrow, with immune-mediated destruction of hematopoietic stem and progenitor cells (HSPCs) playing a central role in its pathogenesis. Although immunosuppressive therapy (IST) and hematopoietic stem cell transplantation (HSCT) have improved survival, a significant proportion of patients remain refractory, relapse after treatment, or lack suitable donors for transplantation. Therefore, novel therapeutic strategies are urgently needed. Chimeric antigen receptor T (CAR-T) cell therapy has demonstrated remarkable efficacy in hematologic malignancies. CD7 is an early surface marker of T-lineage cells and is dispensable for T cell development and function, making it a promising therapeutic target. This exploratory study aims to investigate the molecular and cellular mechanisms of CD7-CAR-T therapy in AA patients by analyzing multi-omics changes before and after treatment. This is a prospective, single-center, single-arm, open-label study enrolling patients with relapsed or refractory severe aplastic anemia. Participants will receive autologous CD7-CAR-T cells following lymphodepletion. Multi-omics profiling, including genomics, transcriptomics, proteomics, and immunophenotyping, will be performed on patient samples before and after infusion. The primary objective is to explore dynamic molecular changes associated with treatment response and disease progression. Secondary objectives include safety evaluation and preliminary assessment of efficacy. Findings from this study may provide mechanistic insights into CD7-CAR-T therapy in AA and inform the development of innovative immunotherapies for bone marrow failure syndromes.

CIBMTR Research Database

The primary purpose of the Research Database is to have a comprehensive source of observational data that can be used to study HSC transplantation and cellular therapies. A secondary purpose of the Research Database is to have a comprehensive source of data to study marrow toxic injuries. Objectives: To learn more about what makes stem cell transplants and cellular therapies work well such as: * Determine how well recipients recover from their transplants or cellular therapy; * Determine how recovery after a transplant or cellular therapy can be improved; * Determine how a donor's or recipient's genetics impact recipient recovery after a transplant or cellular therapy; * Determine how access to transplant or cellular therapy for different groups of patients can be improved; * Determine how well donors recover from the collection procedures.

Exploratory Clinical Study on the Safety and Efficacy of CAR-T Cell Therapy in the Treatment of Relapsed/Refractory Myeloid Malignancies

This is an open-label, single-arm, exploratory clinical trial utilizing a "3+3" dose escalation followed by dose expansion to evaluate the safety, maximum tolerated dose (MTD), pharmacokinetics (PK), and preliminary efficacy of CD33/CD123/CLL-1 CAR-T cell therapy in patients with relapsed/refractory myeloid malignancies. Part A: Dose Escalation Phase. Follows a "3+3" dose escalation design with four predefined dose cohorts: 0.2×10⁶, 0.5×10⁶, 1×10⁶, and 2×10⁶ CAR-positive cells/kg.Anticipated enrollment: 12-24 subjects.Primary objectives: Assess safety, tolerability, and determine MTD.Dose-limiting toxicity (DLT) observation period: 28 days post-infusion. Part B: Dose Expansion Phase.Enrolls 21 additional subjects to receive CAR-T cell infusion at the recommended Phase 2 dose (RP2D) established in Part A.Primary objective: Further evaluate therapeutic efficacy. Overall Study Objectives:Safety profile of CD33/CD123/CLL-1 CAR-T therapy.Efficacy endpoints (e.g., response rates, survival outcomes).Pharmacokinetic characterization of CAR-T cells (expansion/persistence).

EX02 CAR-T Cells for Relapsed and Refractory Acute Myeloid Leukemia

This is a early Phase 1 open-label, single-arm clinical study of EX02 CAR-T therapy for relapsed and refractory acute myeloid leukemia. Each participant will undergo leukapheresis after enrolment, receive treatment of the conditioning chemotherapy of cyclophosphamide and fludarabine, and an an intravenous infusion of CAR-T cells. Each participant will proceed through the following study procedures: * Screening * Enrollment/Leukapheresis * Conditioning chemotherapy * CAR T treatment * Post-treatment assessment * Long-term follow-up