KSV01 Injection as the Therapy for Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia

NCT07246707 | Recruiting Phase 1

• 1 other identifier: KSV01-R102
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single center, single arm, open-label, dose escalation, phase 1 study to evaluate the safety, tolerability and preliminary efficacy of KSV01 injection for patients with relapsed/refractory B-Cell acute lymphoblastic leukemia (r/r B-ALL).

Conditions

B-ALL

Outcome Measures

Primary Outcomes (1)

• Dose-limiting Toxicity

  DLT evaluation period: The DLT evaluation period is defined as within 28 days (inclusive) after the subjects' first administration of KSV01 injection during the dose escalation stage. All adverse events should be graded and evaluated in accordance with CTCAE v5.0. Among them, cytokine release syndrome (CRS) and immune effector cell-related neurotoxicity syndrome (ICANS) should be determined and graded in accordance with the standards of the American Society for Transplantation and Cell Therapy (ASTCT).

  28 days after administration

Study Arms (1)

KSV01 Injection

EXPERIMENTAL

KSV01 Injection is one kind of third-generation non-replicative self-inactivating lentivirus vector.

Drug: KSV01 Injection

Interventions

KSV01 Injection DRUG

KSV01 Injection is one kind of third-generation non-replicative self-inactivating lentivirus vector.

KSV01 Injection

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Voluntary participation and provision of written informed consent by the patient or their legally authorized representative.
• Aged 18 to 80 years (inclusive), any gender.
• Eastern Cooperative Oncology Group (ECOG) performance status of ≤1.
• Life expectancy \> 3 months.
• Diagnosis of B-cell Acute Lymphoblastic Leukemia (B-ALL) according to the 2016 WHO classification, with relapsed/refractory disease defined by meeting at least one of the following criteria:
• Relapse within 12 months of achieving first remission with standard therapy.
• Primary refractory disease: failure to achieve Complete Remission (CR) after two or more cycles of standard chemotherapy.
• Relapsed disease after two or more instances of CR.
• Relapsed or refractory disease following autologous or allogeneic Hematopoietic Stem Cell Transplantation (HSCT).
• Documented CD19-positive leukemia cells in bone marrow or peripheral blood within 1 month prior to screening.
• Morphological disease in the bone marrow (blasts ≥5%).
• For patients with Philadelphia chromosome-positive ALL (Ph+ ALL): must be refractory or intolerant to at least two Tyrosine Kinase Inhibitors (TKIs), including at least one second-generation TKI. Patients with a T315I mutation are exempt from prior TKI salvage therapy.
• Absolute Lymphocyte Count (ALC) ≥ 100/μL.
• Adequate organ function as defined by:
• Hepatic: Alanine aminotransferase (ALT) ≤ 3 × Upper Limit of Normal (ULN); Aspartate aminotransferase (AST) ≤ 3 × ULN; Total bilirubin ≤ 2 × ULN (or ≤ 3 × ULN with a diagnosis of Gilbert's syndrome, with direct bilirubin ≤ 1.5 × ULN).

You may not qualify if:

• Diagnosis of Burkitt's leukemia/lymphoma according to WHO 2016, or chronic myeloid leukemia in accelerated or blast phase.
• History of another primary malignancy that has not been in continuous remission for at least 2 years. Exceptions to the 2-year limit include: non-melanoma skin cancer, curatively treated Stage I solid tumor with low risk of recurrence, cured carcinoma in situ of the cervix (biopsy-confirmed) or squamous intraepithelial lesion on Pap smear, and cured localized prostate cancer.
• Uncontrolled active infection within 4 weeks prior to enrollment.
• Active hepatitis B or hepatitis C virus infection.
• HIV infection.
• Positive for Treponema pallidum(syphilis).
• Severe active autoimmune disease or immunodeficiency, with the exception of well-controlled Type I diabetes and thyroid disorders.
• History of severe allergy or hypersensitivity to macromolecular biological agents (e.g., antibodies, cytokines).
• Participation in another interventional clinical trial within 4 weeks prior to enrollment.
• History of clinically significant central nervous system (CNS) disorders, including but not limited to epilepsy, paresis, aphasia, stroke, severe head injury, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome.
• Central Nervous System (CNS) involvement:
• Presence of CNS 3 disease, defined as detectable blasts in the cerebrospinal fluid (CSF) with ≥5 WBCs/mm³, with or without neurological symptoms.
• Presence of CNS 2 disease, defined as detectable blasts in the CSF with \<5 WBCs/mm³ AND the presence of neurological symptoms.
• Note: Subjects with CNS 1 status (no detectable leukemic blasts in CSF) and subjects with CNS 2 status without significant clinical neurological abnormalities are eligible.
• History or presence of any CNS disorder, such as seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, any autoimmune disorder involving the CNS, posterior reversible encephalopathy syndrome, or cerebral edema.

Sponsors & Collaborators

• Zhejiang University: lead
• TCRx Therapeutics Co.Ltd: collaborator

Study Sites (1)

First Affiliated Hospital of Zhejiang University

Hangzhou, China

RECRUITING

Study Officials

• He Huang, MD

  First Affiliated Hospital of Zhejiang University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

He Huang, MD

CONTACT

057187233772; hehuangyu@126.com

Yongxian Hu, MD

CONTACT

057187233772; huyongxian2000@aliyun.com

Study Design

• Study Type: interventional
• Phase: 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: November 17, 2025
• First Posted: November 24, 2025
• Study Start: November 1, 2025
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): June 1, 2028
• Last Updated: November 24, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)