Clinical trials

The purpose of this study is to evaluate the efficacy and safety of glofitamab in combination with gemcitabine plus oxaliplatin (GemOx) versus standard of care (SOC) in patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) who have relapsed early (within 1 year) or are primary refractory to first-line therapy. Participants will be randomly assigned in a 1:1 ratio to receive either the Glofitamab-GemOx combination regimen or SOC. The SOC arm consists of investigator's choice of salvage chemoimmunotherapy followed by autologous stem cell transplantation (ASCT) for eligible patients. The primary endpoint of the study is event-free survival (EFS).

Venetoclax combined with azacitidine (VEN-AZA) is the current first-line standard of care for newly diagnosed acute myeloid leukemia (AML) patients unfit for intensive chemotherapy. Although this regimen substantially improves remission rates, marked inter-individual variability is observed in clinical practice-ranging from severe myelosuppression or tumor lysis syndrome in some patients to poor response or early relapse in others. Venetoclax is primarily metabolized by CYP3A4, and its systemic exposure is modulated by multiple factors, including hepatic and renal function, concomitant medications (particularly azole antifungals), and UGT1A1 polymorphisms, leading to a 50%-70% inter-individual variability in blood drug concentrations. Despite this variability, the current VEN-AZA regimen employs a fixed-dose strategy (400 mg/day) without incorporating therapeutic drug monitoring (TDM) to guide individual dosing. Critical knowledge gaps remain: (1) whether a clear exposure-response relationship exists between venetoclax exposure and composite remission rate (CR+CRi); (2) what blood concentration range optimizes efficacy while minimizing toxicity; (3) which covariates significantly influence venetoclax clearance; and (4) whether early concentration sampling can reliably predict subsequent exposure and clinical outcomes.\* To address these questions, investigators designed a prospective study enrolling newly diagnosed AML patients receiving VEN-AZA therapy. Investigators aim to systematically characterize the exposure-response relationship, establish an optimal therapeutic concentration window, identify key covariates contributing to inter-individual pharmacokinetic variability, and evaluate early-sampling prediction strategies. The findings are expected to provide direct evidence for TDM-guided individualized dosing and to support a paradigm shift from a "fixed-dose" to a "concentration-guided" approach in precision AML therapy.

Hematopoietic stem cell transplantation is an important therapeutic approach for hematologic malignancies, and the impact of donor age on transplant outcomes remains an active area of investigation. Older donors may be associated with impaired stem cell fitness, delayed immune reconstitution, and reduced T-cell function. However, randomized controlled trials directly comparing transplant outcomes by donor age are difficult to conduct because of ethical constraints, and previous retrospective studies have yielded inconsistent findings due to confounding bias and limited causal interpretability. Target trial emulation (TTE) is a methodological framework that uses observational data to emulate the design principles of a randomized trial, thereby reducing biases such as immortal time bias, time-varying confounding, and prevalent-user bias, and improving the validity of causal inference. Therefore, this study will use a large single-center retrospective clinical cohort to perform a TTE analysis, aiming to approximate the causal framework of an randomized trial and systematically evaluate the effect of donor age on clinical outcomes after allo-HSCT, thereby providing higher-quality evidence to optimize donor selection strategies.

This clinical trial aims to compare the efficacy and safety of venetoclax-based consolidation therapy versus conventional consolidation chemotherapy (intermediate/high-dose cytarabine) in newly diagnosed adult patients with intermediate-risk acute myeloid leukemia (AML). Participants must have achieved complete remission (CR) or CR with incomplete hematologic recovery (CRi) after induction therapy with venetoclax and azacitidine and are planned to undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT).

The goal of this clinical trial is to compare the efficacy and safety of a venetoclax-based consolidation therapy versus conventional consolidation chemotherapy in newly diagnosed adult patients with high-risk acute myeloid leukemia (AML) who have achieved complete remission (CR) or CR with incomplete hematologic recovery (CRi) after induction therapy with venetoclax and azacitidine and are planned for transplantation. The main questions it aims to answer are: Does consolidation therapy with a venetoclax-containing regimen lead to superior clinical outcomes compared to conventional chemotherapy in this specific patient population? What is the comparative safety profile of the venetoclax-containing consolidation regimen versus conventional chemotherapy in these patients? Participants will be randomly assigned to receive either the venetoclax-based consolidation therapy or the conventional consolidation chemotherapy before undergoing transplantation.

In endoscopic treatment for gastric submucosal tumors arising from the muscularis propria, does robot-assisted surgery yield a higher serosa preservation rate, shorter operative time and lower complication rates than conventional endoscopic procedures?

This observational study aims to emulate a hypothetical pragmatic, multicenter, non-blinded clinical trial enrolling adult patients undergoing haploidentical hematopoietic stem cell transplantation (haplo-HSCT) with grafts derived from either cousin or sibiling donors. The main research question is whether cousin donor-derived haplo-HSCT is associated with inferior clinical outcomes relative to sibling donor-derived haplo-HSCT. Study participants consist of adult patients diagnosed with hematological malignancies who underwent cousin or sibiling haplo-HSCT between 2017 and 2024, and all eligible recipients will receive regular follow-up assessments.

This is an Open, Prospective, Single-arm Study, which is designed to evaluate the efficacy, safety and pharmacokinetics of ThisCART19A Combined With Olverembatinib for the treatment of Newly Diagnosed Ph-positive lymphoblastic leukemia.

This is a single-arm, dose-escalation exploratory study evaluating the safety and efficacy of RN1201, a BCMA/CD19-targeted allogeneic CAR-T cell therapy, in patients with newly diagnosed multiple myeloma. Patients will receive lymphodepletion followed by a single infusion of RN1201. Primary endpoints include incidence and severity of treatment-emergent adverse events. Secondary endpoints assess response rate and minimal residual disease (MRD) status.

This single-arm, open-label exploratory trial aims to evaluate the safety, feasibility, and preliminary efficacy of RN1201 Injection in patients with antibody-mediated diseases, including refractory immune-mediated platelet transfusion refractoriness (PTR) and relapsed or refractory immune thrombocytopenia (ITP). Patients will receive RN1201 cells infusion following lymphodepletion. The study will assess safety, response rates, B-cell depletion, and immune reconstitution. Exploratory analyses will examine in vivo persistence and activity of RN1201.

This study is a response-adapted, multicenter, interventional trial enrolling patients with HER2-positive early or locally advanced breast cancer. All enrolled patients will first receive 2 cycles of standard neoadjuvant THP regimen, consisting of a taxane, trastuzumab, and pertuzumab. After the initial 2-cycle treatment, tumor response will be evaluated by radiologic imaging and patient-derived organoid (PDO) drug sensitivity testing. Patients with an inadequate response to THP are defined as those with \<50% tumor size reduction on imaging, or failure to reach the PDO sensitivity threshold (\<80% tumor cell killing for HER2+/HR- tumors; \<60% for HER2+/HR+ tumors). These non-responders will switch to receive 4 cycles of trastuzumab rezetecan (SHR-A1811), a novel HER2-targeted antibody-drug conjugate (ADC). Patients with a favorable response (≥50% tumor reduction or meeting the PDO threshold) will continue with an additional 4 cycles of THP. The primary objective is to evaluate the pathologic complete response (pCR) rate in THP non-responders after switching to trastuzumab rezetecan. Secondary objectives include objective response rate (ORR), event-free survival (EFS), overall survival (OS), 3-year invasive disease-free survival (iDFS), and safety profiles of both treatment strategies. Outcomes in patients who continue THP will be described for exploratory purposes. A total of 124 patients will be enrolled. This response-adapted, individualized strategy aims to provide an effective option for HER2-positive breast cancer patients with an inadequate early response to conventional THP neoadjuvant therapy.

This study was designed as a prospective, multicenter, open-label, randomized controlled trial. Eligible participants were patients aged 15-65 years with high risk or relapsed/refractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic neoplasms (MDS), diagnosed based on bone marrow morphology, immunophenotyping, genetic testing, and treatment response assessment. The experimental group received SHR2554 combined with azacitidine as an overlapped sequential combination with the mBuCy conditioning regimen, whereas the control group received the mBuCy conditioning regimen, both followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT). The primary endpoint is 1-year event-free survival (EFS). Secondary endpoints include 2-year overall survival, 2-year cumulative incidence of relapse, transplant-related mortality, incidence of acute/chronic GVHD, and safety profiles.

The purpose of this study is to explore the efficacy and safety of venetoclax combined with azacitidine（VA) induction followed by bridging allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of hypoplastic and secondary acute myeloid leukemia (AML).

Platelet transfusion refractoriness (PTR) is a common complication in patients with hematological malignancies. It not only prolongs the duration of platelet transfusion dependence and significantly increases the risk of bleeding, but is also strongly associated with graft failure and reduced survival after transplantation. HLA class I antibody-mediated alloimmunization is recognized as the most important immunological cause of PTR. HLA antibodies are directly secreted by plasma cells, which are derived from B cells. Therefore, targeting B cells to reduce antibody production is a crucial step in eliminating HLA antibodies. Bruton's tyrosine kinase (BTK) is expressed throughout B cell development from the pre-B cell stage to maturity and supports B cell development, maturation, survival, proliferation, and antibody production by acting as a downstream kinase in the B cell receptor signaling pathway. Bortezomib, a proteasome inhibitor, can selectively induce apoptosis in long-lived plasma cells. The investigators' preliminary exploratory use of a BTK inhibitor in the treatment of PTR with HLA antibodies significantly reduced the mean fluorescence intensity (MFI) of HLA antibodies, improved platelet transfusion outcomes, and demonstrated a favorable safety profile. Based on these findings, the investigators are conducting a prospective, multicenter, randomized controlled two-arm study to investigate the efficacy and safety of acalabrutinib and bortezomib in eliminating HLA antibodies in hematological malignancies patients with PTR.

To evaluation the efficacy and safety of autologous fecal bacteria transplantation in preventing acute graft versus host disease after haploidentical hematopoietic stem cell transplantation. Bone marrow transplant patients were recruited.

With the stem cell transplanting increasing, patients which effected with gut GVHD were also increased. To evaluation the safety and efficacy of FMT for gut GVHD,patients with gut GVHD were recruited.

This study is a single-arm, open-label clinical investigation to evaluate the tolerance, safety and preliminary efficacy of CAR-T (U96) in the treatment of relapsed/refractory B-cell tumors. The study will be conducted in two disease types, acute B-lymphoblastic leukemia and B-cell lymphoma, with a dose escalation plan using the "3+3" method. Each dose group is planned to enroll 3 to 6 patients, with a total of approximately 30 to 48 patients to be enrolled in the entire study. After signing the informed consent form, patients will undergo screening tests. If they meet the inclusion and exclusion criteria, they will be enrolled in the study. After receiving U96 treatment, patients will be followed up. It is recommended that they stay in the hospital for at least 14 days after administration. Safety and efficacy follow-ups will be conducted at 28 days and 3, 6, 12, 18, and 24 months after treatment. The follow-up period after treatment will last for 2 years, with a long-term follow-up of 15 years to assess the efficacy and safety until the end of the study or the patient withdraws from the study. For patients who have received U96 treatment, even if they withdraw from the study early, the investigators should still conduct long-term safety follow-ups according to the protocol to evaluate the long-term safety of the product.

A Phase II Study of Pomalidomide Combined with Anti-CD20 Monoclonal Antibody and Prednisone in Frontline Indolent B-Cell Lymphoma Objective: This prospective, single-arm, Phase II trial aims to evaluate the efficacy and safety of first-line pomalidomide plus anti-CD20 antibody and prednisone in patients with indolent B-cell lymphoma. Study Population: Approximately 30 adult patients (age ≥18 years) will be enrolled. Eligible histologies include follicular lymphoma (FL), CD20-positive marginal zone lymphoma (MZL: extranodal MALT, splenic SMZL, nodal NMZL), indolent mantle cell lymphoma (MCL), chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), and lymphoplasmacytic lymphoma/Waldenström macroglobulinemia. Patients must be treatment-naïve, have an indication for systemic therapy, ECOG performance status 0-2, and adequate bone marrow reserve (ANC ≥1.5×10⁹/L, platelets ≥75×10⁹/L, hemoglobin ≥9.0 g/dL; lower thresholds permitted if marrow/spleen involvement, per investigator discretion). Adequate organ function is required: bilirubin ≤2×ULN, ALT/AST ≤2.5×ULN, and creatinine clearance \>30 mL/min. Life expectancy must be ≥3 months, and written informed consent is mandatory. Exclusion Criteria: Patients are excluded if they have another malignancy within 5 years (unless curatively treated without recurrence), CNS lymphoma involvement, transformation to high-grade lymphoma, uncontrolled infection, severe comorbidities affecting study participation, significant non-lymphoma-related organ dysfunction (ALT/AST \>3×ULN, bilirubin \>2×ULN, creatinine \>1.5×ULN), active CNS dysfunction, major surgery within 30 days, pregnancy or lactation, lack of contraception in women of childbearing potential, known drug hypersensitivity, or any condition deemed unsuitable by the investigator. Treatment Regimen: Induction consists of six 28-day cycles. Anti-CD20 antibody is administered at 375 mg/m² weekly during Cycle 1 and on Day 1 of Cycles 2-6. Pomalidomide is given at 4 mg/day on Days 2-22 of Cycles 1-6. Prednisone is administered at 100 mg/day on Days 1-5 of Cycles 1-6. Maintenance therapy continues for 2 years with pomalidomide 4 mg/day on Days 1-14 and anti-CD20 antibody 375 mg/m² on Day 1 every 8 weeks. Endpoints: The primary endpoint is overall response rate (ORR). Secondary endpoints include complete response rate (CR), progression-free survival (PFS), overall survival (OS), and safety (hematologic and non-hematologic adverse events). Statistical Methods: Continuous variables will be summarized with descriptive statistics; categorical variables with frequencies and percentages. Time-to-event endpoints (PFS, OS, and duration of response) will be analyzed using the Kaplan-Meier method, reporting medians, quartiles, and 90% confidence intervals, along with event and censoring counts. ORR will be tested statistically and reported with a 90% confidence interval. Timeline: The study is expected to begin in January 2026, complete enrollment by December 2026, and conclude by December 2027. The total planned sample size is 30 patients.

This study is a prospective, multicenter, open-label, single-arm cohort study to evaluate the effectiveness and safety of romiplostim in patients with immune thrombocytopenia (ITP) who have not responded to oral thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag, hetrombopag, or avatrombopag. ITP is a blood disorder in which the immune system attacks and destroys the body's own platelets, leading to low platelet counts and an increased risk of bleeding. While oral TPO-RAs are effective for many patients, approximately 20-30% of patients do not respond adequately or lose response over time. For these patients, alternative treatments are urgently needed. Participants in this study will discontinue their current oral TPO-RA and receive romiplostim as a weekly subcutaneous injection. The starting dose is 3 µg/kg, and the dose may be adjusted weekly based on platelet counts (maximum 10 µg/kg). The total treatment and follow-up period is 24 weeks. The primary outcome measure is the durable platelet response rate at week 24, defined as maintaining platelet counts at ≥50×10⁹/L for at least two scheduled visits during weeks 22-24 without rescue therapy. Secondary outcomes include the sustained response rate at week 12, complete response rate at week 24, time to first response, improvement in bleeding events, and quality of life assessment. Safety outcomes include monitoring for thromboembolic events, bone marrow fibrosis, hepatotoxicity, injection site reactions, and other adverse events according to CTCAE v5.0. Approximately 60 participants will be enrolled across multiple centers in China. This study aims to provide evidence for romiplostim as a treatment option for ITP patients who have failed oral TPO-RAs.

The purpose of this study is to evaluate the efficacy and safety of glofitamab in combination with gemcitabine plus oxaliplatin (GemOx) versus standard of care (SOC) in patients with relapsed/refractory large B-cell lymphoma (R/R LBCL) who have relapsed early (within 1 year) or are primary refractory to first-line therapy. Participants will be randomly assigned in a 1:1 ratio to receive either the Glofitamab-GemOx combination regimen or SOC. The SOC arm consists of investigator's choice of salvage chemoimmunotherapy followed by autologous stem cell transplantation (ASCT) for eligible patients. The primary endpoint of the study is event-free survival (EFS).

This is an open-label, multi-center, single-arm clinical study evaluating the efficacy and safety of the VABu conditioning regimen in elderly patients (≥60 years) with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (HSCT). The VABu regimen consists of Venetoclax, Azacitidine, Semustine, Cytarabine, and Busulfan. All enrolled participants will receive the VABu regimen as conditioning therapy prior to HSCT. The study aims to enroll 20 participants from multiple centers in China. The primary objectives are to evaluate the overall response rate, cumulative relapse rate, overall survival, graft-versus-host disease (GVHD)-free relapse-free survival (GRFS), non-relapse mortality (NRM), incidence of acute and chronic GVHD, and reactivation rates of cytomegalovirus (CMV) and Epstein-Barr virus (EBV). Safety outcomes include treatment-related toxicities, such as bone marrow suppression, infection, and organ dysfunction.

This is a multi-center, prospective, open-label randomized controlled trial evaluating the efficacy and safety of virtual reality-based mindfulness-based cognitive therapy (VR-MBCT) as an adjunct to usual care in adults with chronic nonspecific low back pain (CNLBP). A total of 214 participants aged 20-70 years with pain duration ≥3 months and average back pain bothersomeness ≥4 (0-10 scale) will be enrolled from three hospitals in China and randomized 1:1 to an 8-week VR-MBCT intervention group or a waitlist usual care control group. The co-primary outcomes are the proportions of patients achieving ≥30% improvement from baseline in Oswestry Disability Index (ODI) and self-rated back pain bothersomeness at week 8. Secondary outcomes include pain intensity (NRS), global improvement, anxiety (GAD-7), depression (PHQ-9), physical activity (IPAQ-SF), sleep quality (SQS), fear-avoidance beliefs (FABQ-PA), pain catastrophizing (PCS), work productivity (WPAI-GH), treatment use, system usability (SUS), and patient satisfaction. Safety will be monitored for VR-related adverse events. Assessments will be performed at baseline (T0), week 4 (T1), week 8 (T2), and week 26 (T3). Data will be analyzed following the intention-to-treat principle using mixed-effects models and per-protocol analyses.

Objectives: This study aims to evaluate the clinical efficacy and safety of repetitive transcranial magnetic stimulation (rTMS) in patients with diarrhea-predominant irritable bowel syndrome (IBS-D), and to explore the potential underlying mechanisms by which rTMS alleviates clinical symptoms in IBS-D patients. Design: This is a clinical trial that uses clinical symptom scales to assess the therapeutic effect of rTMS on IBS-D patients. Meanwhile, gut microbiota and metabolite profiling, as well as the methane-hydrogen breath test, will be applied to investigate the mechanism of action from the perspectives of gut microecology, intestinal motility, and metabolism, so as to provide scientific evidence for the clinical application of rTMS in the treatment of IBS-D.

This is a prospective, open-label, single-arm, two-cohort Phase 2 clinical study designed to evaluate the efficacy and safety of Bcl-2 Inhibitor combined with azacitidine (with blinatumomab added in B/myeloid subtype) in patients with newly diagnosed mixed phenotype acute leukemia (MPAL). Eligible subjects are divided into two cohorts based on immunophenotype: Cohort A (T/Myeloid MPAL) receives Bcl-2 Inhibitor + azacitidine, and Cohort B (B/Myeloid MPAL) receives Bcl-2 Inhibitor + azacitidine + blinatumomab. The treatment cycle is 28 days, with the primary efficacy endpoint assessed after 2 cycles of induction therapy. Patients who achieve CRc will undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT) following 2 to 3 cycles of consolidation therapy.The total enrollment period is 24 months, and all subjects will be followed up for at least 24 months from the first day of the first cycle (C1D1). The primary objective is to evaluate the composite complete response (CRc) rate after 2 cycles of induction therapy , and the secondary objectives include evaluating measurable residual disease (MRD) negativity rate, bridge-to-allogeneic hematopoietic stem cell transplantation (allo-HSCT) rate in first complete response (CR1), overall survival(OS),Event-Free Survival(EFS),Relapse-Free Survival(RFS) and Safety.

This observational study aims to evaluate the diagnostic value and clinical utility of detecting the KIT-D816V mutation in the peripheral blood of adult patients with systemic mastocytosis (SM), using droplet digital PCR (ddPCR). Currently, the diagnosis of SM relies heavily on invasive bone marrow biopsies. This study will determine whether highly sensitive ddPCR testing of peripheral blood could provide a reliable, minimally invasive alternative for detecting the KIT-D816V mutation, which is a key driver of the disease and a major diagnostic criterion. The results could optimize the diagnostic process and continuous monitoring of adult SM patients.