Clinical Study of SYNCAR-100 in the Treatment of Relapsed/Refractory Acute B-Lymphoblastic Leukemia

NCT07429461 | Recruiting Early Phase 1

• 1 other identifier: BSW-001-I
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to assess the safety, tolerability, and preliminary efficacy of SYNCAR-100 in patients with CD19-positive relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL). Participants who have signed the informed consent form will undergo screening against the inclusion and exclusion criteria. Eligible participants will receive study drug administration once weekly for a total of four doses, followed by a 1-year safety and efficacy follow-up observation period. After the completion of the study, long-term follow-up may be required for participants to monitor their health and survival status until 15 years post-treatment, or until the occurrence of patient death, loss to follow-up, or withdrawal of consent.

Conditions

B-Cell Acute Lymphoblastic Leukemia

Keywords

B-Cell Acute Lymphoblastic Leukemia; Relapsed Refractory Leukemia; CD19 Antigens; Chimeric Antigen Receptor T-Cells; circRNA; Nucleic Acids; Drug Safety; Clinical Study

Outcome Measures

Primary Outcomes (1)

• Adverse events（AE）

  Evaluated through laboratory investigations, 12-lead electrocardiography, and vital signs.

  within 48 weeks post-dose

Secondary Outcomes (8)

• Overall Response Rate（ORR）

  within 12 weeks and 48 weeks post-dose

• Minimal Residual Disease（MRD）

  within 12 weeks and 48 weeks post-dose

• Duration of Response（DOR）

  within 12 weeks and 48 weeks post-dose

• Progression-Free Survival（PFS）

  within 12 weeks and 48 weeks post-dose

• Overall Survival（OS）

  within 12 weeks and 48 weeks post-dose

Study Arms (1)

SYNCAR-100 Treatment Group

EXPERIMENTAL

Weekly intravenous (IV) administration for 4 doses. The investigator and sponsor may determine the dosing interval and number of doses for subsequent dose cohorts based on the safety/tolerability, pharmacokinetic parameters, clinical efficacy and prior clinical study experience of the 0.02 mg/kg dose cohort.

Drug: SYNCAR-100 Injection

Interventions

SYNCAR-100 Injection DRUG

Subjects will receive intravenous injections once weekly for a total of 4 injections.

SYNCAR-100 Treatment Group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged 18 to 75 years (inclusive), of any gender.
• Karnofsky Performance Status score ≥ 70.
• Estimated life expectancy ≥ 12 weeks.
• Positive CD19 expression on tumor cells confirmed by flow cytometry in bone marrow or peripheral blood.
• Confirmed diagnosis of B-cell acute lymphoblastic leukemia (B-ALL) by bone marrow examination, and meeting one of the following criteria:
• ① Refractory B-ALL: Failure to achieve complete remission (CR) after 2 courses of standard induction chemotherapy, or failure to achieve CR after first-line/multiline salvage chemotherapy.
• ② Relapsed B-ALL: Relapse within 12 months after the first remission, or relapse after first-line/multiline salvage chemotherapy.
• ③Relapse after autologous or allogeneic hematopoietic stem cell transplantation (HSCT).
• ④ For Philadelphia chromosome-positive (Ph+) patients: At least 2 lines of tyrosine kinase inhibitor (TKI) therapy have failed, or the patient is intolerant to TKI therapy, or the patient harbors the T315I mutation and is resistant to TKIs.
• Proportion of blasts and immature lymphocytes in bone marrow \> 5% confirmed by bone marrow morphologic examination.
• No prior hematopoietic stem cell transplantation (HSCT) within 6 months before enrollment.
• Adequate organ function reserve, as defined by all of the following:
• ① Creatinine clearance (calculated by the Cockcroft-Gault formula) \> 60 mL/min: Male: Creatinine Clearance = \[(140 - Age) × Body Weight (kg)\] / \[0.818 × Serum Creatinine (μmol/L)\] Female: Creatinine Clearance = \[(140 - Age) × Body Weight (kg) × 0.85\] / \[0.818 × Serum Creatinine (μmol/L)\]
• ② Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \< 2.5 times the upper limit of normal (ULN) ; for patients with hepatic metastasis, AST and ALT ≤ 5 times the upper limit of normal (ULN) .
• ③Serum total bilirubin \< 1.5 times the upper limit of normal (ULN) ; for patients with Gilbert's syndrome, total bilirubin ≤ 3 times the upper limit of normal (ULN) .

You may not qualify if:

• Isolated extramedullary leukemia or isolated extramedullary relapse.
• Central nervous system (CNS) involvement of leukemia at CNS2 stage.
• A history of other malignant tumors, except for the following: cured non-melanoma skin cancer, carcinoma in situ of the uterine cervix, localized prostate cancer, superficial bladder cancer, ductal carcinoma in situ, or other malignant tumors with disease-free survival for more than 5 years.
• Positive test results for any of the following infectious diseases: HIV; HCV; HBsAg; positive HBcAb with concurrent positive HBV DNA copy number; TPPA.
• Administration of live or attenuated live vaccines within 4 weeks prior to enrollment.
• Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) with tyrosine kinase inhibitor (TKI) therapy within 1 week prior to enrollment.
• Prior receipt of CD19-targeted therapy, CAR-T cell therapy, or other gene-edited T cell therapy.
• Grade ≥2 acute graft-versus-host disease (GVHD) (per Glucksberg criteria) requiring treatment, or extensive chronic GVHD (per Seattle criteria) within 4 weeks prior to enrollment; or patients judged by the investigator to potentially require anti-GVHD therapy during the study period.
• Comorbidities judged by the investigator to require systemic corticosteroid therapy or other immunosuppressive therapy during the study period; or receipt of allogeneic cellular therapy (e.g., donor lymphocyte infusion \[DLI\]) within 4 weeks prior to enrollment.
• Receipt of CNS-directed radiotherapy within 4 weeks prior to enrollment.
• Unresolved acute toxicities from prior therapy (excluding hematologic toxicities and alopecia) that have not recovered to Grade 1 or lower.
• Known life-threatening hypersensitivity or other intolerance to the study drug, or a history of severe atopy.
• Active autoimmune diseases (including but not limited to systemic lupus erythematosus, Sjögren's syndrome, rheumatoid arthritis, psoriasis, multiple sclerosis, inflammatory bowel disease, Hashimoto's thyroiditis, etc.), excluding hypothyroidism that is controllable solely with hormone replacement therapy.
• Receipt of major surgery requiring general anesthesia within 4 weeks prior to enrollment; or failure to recover and achieve clinical stability from prior surgical treatment; or anticipated major surgery requiring general anesthesia during the study period.
• Use of other investigational drugs within 28 days prior to enrollment.

Sponsors & Collaborators

• Zhejiang University: lead
• Bisheng (Beijing) Biotechnology Co., Ltd.: collaborator

Study Sites (1)

The first affiliated hospital of medical college of zhejiang university

Hangzhou, Zhejiang, 310003, China

RECRUITING

MeSH Terms

Conditions

Burkitt Lymphoma; Leukemia

Condition Hierarchy (Ancestors)

Epstein-Barr Virus Infections; Herpesviridae Infections; DNA Virus Infections; Virus Diseases; Infections; Tumor Virus Infections; Lymphoma, B-Cell; Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Hematologic Diseases

Central Study Contacts

He Huang, MD

CONTACT

13605714822; hehuangyu@126.com

Yongxian Hu, MD

CONTACT

057187233772; huyongxian2000@aliyun.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: February 4, 2026
• First Posted: February 24, 2026
• Study Start: February 28, 2026
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2041
• Last Updated: February 24, 2026

Record last verified: 2026-02

Locations

CN (1)