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

Recruiting now · Phase 1

Conditions studied: B-ALL

In brief

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).

Key facts

Study ID

NCT07246707

Run by

Zhejiang University

People needed

30

Starts

2026-01-02

Expected to finish

2028-06-01

Last updated by the study team

2026-07-17

Who can join

Age: 18 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

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).
• Renal: Creatinine clearance (calculated by Cockcroft-Gault formula) ≥ 60 mL/min.
• Pulmonary: Oxygen saturation (SaO2) ≥ 92% on room air, and no active pulmonary infection.
• Cardiac: Left Ventricular Ejection Fraction (LVEF) ≥ 40% by echocardiography; absence of significant pericardial effusion; no clinically significant electrocardiogram (ECG) abnormalities.
• For women of childbearing potential: negative urine or serum pregnancy test at screening, and agreement to use effective contraception for at least 1 year post-infusion. Male subjects with partners of childbearing potential must agree to use effective barrier contraception for at least 1 year post-infusion.
• For subjects with prior blinatumomab (CD3-CD19 bispecific T-cell engager) therapy: CD19 tumor expression on blasts (from bone marrow or peripheral blood) must be documented after the most recent cycle of blinatumomab. If CD19 expression is quantified, the percentage of CD19-positive blasts must be ≥90%.

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.
• History of concomitant genetic syndromes associated with bone marrow failure, such as Fanconi anemia, Kostmann syndrome (severe congenital neutropenia), Shwachman-Diamond syndrome.
• History of any of the following cardiovascular conditions within the past 6 months: New York Heart Association (NYHA) Class III or IV heart failure, cardiac angioplasty or stenting, myocardial infarction, unstable angina, or other clinically significant cardiac disease.
• Active psychiatric illness.
• History of drug abuse/addiction.
• Use of the following medications or therapies:
• Salvage systemic therapy (including chemotherapy, TKIs for Ph+ ALL, and blinatumomab) within 1 week or 5 half-lives (whichever is shorter) prior to study drug infusion.
• Note:
• TKIs and hydroxyurea must be discontinued at least 72 hours prior to study drug infusion.* 6-mercaptopurine, 6-thioguanine, methotrexate (standard dose), cytarabine (standard dose), vincristine, and asparaginase must be discontinued at least 1 week prior.* Intrathecal chemotherapy for CNS prophylaxis must be discontinued at least 1 week prior.* PEG-asparaginase must be discontinued at least 4 weeks prior.*
• Prior anti-CD19 therapy other than blinatumomab.
• History of Grade 4 neurological toxicity (per CTCAE v6.0) or Grade 4 CRS (per Lee 2014 criteria) during prior blinatumomab treatment.

Where it is running

• First Affiliated Hospital of Zhejiang University — Hangzhou, China (enrolling)

Full record on ClinicalTrials.gov

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