B-ALLPhase 1NCT07246707
KSV01 Injection as the Therapy for Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia
A Phase 1 study testing KSV01 Injection in people with B-ALL, run by Zhejiang University at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- B-ALL
- What is being tested?
- KSV01 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 2 Jan 2026to Jun 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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%.
Who cannot join
- 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).
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- 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.
- Prior treatment with alemtuzumab within 6 months, or with clofarabine or cladribine within 3 months prior to study drug infusion.
- Systemic treatment for Graft-versus-Host Disease (e.g., calcineurin inhibitors, methotrexate, mycophenolate mofetil, sirolimus, thalidomide) or immunosuppressive antibody therapy (e.g., anti-CD20, anti-TNF, anti-IL-6, or anti-IL-6R antibodies) within 4 weeks prior to enrollment.
- Any prior systemic therapy with inhibitory/stimulatory immune checkpoint molecules (e.g., ipilimumab, nivolumab, pembrolizumab, atezolizumab, OX40 agonists, 4-1BB agonists). A washout period of at least 3 half-lives from the last dose is required before enrollment.
- Radiotherapy: Non-CNS directed radiotherapy within 2 weeks or CNS-directed radiotherapy within 8 weeks prior to study drug infusion.
- Corticosteroids: Therapeutic doses of corticosteroids (defined as prednisone equivalent >20 mg/day) within 72 hours prior to study drug infusion. Physiologic replacement doses, and topical or inhaled steroids are permitted.
- Prior gene therapy.
- Acute Graft-versus-Host Disease (GVHD) of Grade II to IV per Glucksberg criteria, or overall grade B-D per the IBMTR Severity Index; OR acute or chronic GVHD requiring systemic therapy within 4 weeks prior to enrollment.
- Administration of a live vaccine within 4 weeks prior to enrollment.
- Pregnancy or lactation.
- Any condition that, in the investigator's judgment, may compromise the subject's ability to complete all required study visits and procedures (including follow-up), or comply with the study requirements.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
First Affiliated Hospital of Zhejiang University
Hangzhou
He Huang, MD/PhD, +（86）0571-87236703
About this study
Official title: A Phase I Clinical Study on the Safety, Tolerability, and Efficacy of KSV01 Injection in Patients With Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia
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).
Read the detailed description
This investigator-initiated clinical study aims to evaluate KSV01 injection, the third-generation self-inactivating lentiviral vector that carries CD19 CAR, in patients with r/r B-ALL. The study employs a dose escalation design to assess safety, tolerability, and preliminary efficacy.
- Conditions
- B-ALL
- Being tested
- KSV01 Injection
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KSV01 InjectionDrug
KSV01 Injection is one kind of third-generation non-replicative self-inactivating lentivirus vector.
| Study group | Role | What they receive |
|---|---|---|
| KSV01 Injection | Experimental | KSV01 Injection is one kind of third-generation non-replicative self-inactivating lentivirus vector. Receives: Drug: KSV01 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 v6.0. 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).
Measured: 28 days after administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2025
- Study start2 Jan 2026
- Primary completionOct 2027 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for B-ALLStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhejiang University
- Sponsor type
- University, hospital or other
- Collaborators
- TCRx Therapeutics Co.Ltd
- Responsible party
- Principal investigator, He Huang, Zhejiang University
- Investigators
- He Huang, MD, Principal investigator, First Affiliated Hospital of Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07246707
- Sponsor's protocol ID
- KSV01-R102
- Status last verified
- Oct 2025
- First submitted
- 17 Nov 2025
- Last updated
- 17 Jul 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Clinical Study on the Safety, Tolerability, and Efficacy of KSV01 Injection in Patients With Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia
- ClinicalTrials.gov ID
- NCT07246707
- Acronym / sponsor ID
- KSV01-R102
- Conditions
- B-ALL
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Zhejiang UniversityUniversity, hospital or other
- Collaborators
- TCRx Therapeutics Co.Ltd
- Study start
- 2 Jan 2026 (actual)
- Primary completion
- Oct 2027 (estimated)
- Study completion
- Jun 2028 (estimated)
- First posted
- 24 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
This investigator-initiated clinical study aims to evaluate KSV01 injection, the third-generation self-inactivating lentiviral vector that carries CD19 CAR, in patients with r/r B-ALL. The study employs a dose escalation design to assess safety, tolerability, and preliminary efficacy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| KSV01 Injection | Experimental | Drug: KSV01 InjectionKSV01 Injection is one kind of third-generation non-replicative self-inactivating lentivirus vector. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- 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:
Key exclusion criteria (35 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KSV01 InjectionKSV01 Injection is one kind of third-generation non-replicative self-inactivating lentivirus vector. | Drug | KSV01 Injection |
Primary outcome
Dose-limiting Toxicity
Time frame: 28 days after administration
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 v6.0. 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).
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
He Huang, MD principal investigator
First Affiliated Hospital of Zhejiang University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing KSV01 Injection in people with B-ALL, run by Zhejiang University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 20 inclusion and 35 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact He Huang, MD (057187233772, hehuangyu@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose-limiting Toxicity (time frame: 28 days after administration). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jul 2026; AzTrials last refreshed its copy on 31 Oct 2026.