Transfusion-dependent Beta-ThalassemiaPhase 1/2NCT06280378
β-Thalassemia Treatment With KL003 Cell Injection
A Phase 1/2 study testing KL003 Cell Injection Drug Product in people with Transfusion-dependent Beta-Thalassemia, run by Kanglin Biotechnology (Hangzhou) Co., Ltd. at 2 sites in China.
Trial summary
This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with β-thalassemia major. Read more about this study
- Who is it for?
- Ages 3 to 35 years
- Conditions the study is for
- Transfusion-dependent Beta-Thalassemia
- What is being tested?
- KL003 Cell Injection Drug Product
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 2 sitesChina
- Study dates
- 5 Apr 2024to 20 Dec 2028
- Phase
- Phase 1/2
- 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 3 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female age between 3-35 years;
- Diagnosis of transfusion-dependent β-thalassemia and a history of at least 100 mL/kg/year of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years;
- Karnofsky performance status ≥70 for participants≥16 years of age; Lansky performance status of ≥70 for participants<16 years of age;
- Eligible to undergo auto-HSCT;
- Willing and able to follow the research procedures and conditions, with good compliance;
- Willing to receive at least the 2 years follow-up;
- Participant and/or legal guardians voluntarily participated in this clinical trial and signed the informed consent form.
Who cannot join
- Diagnosis of composite α thalassemia;
- Prior receipt of gene therapy or allo-HSCT;
- Meet the criteria for allo-HSCT and with an identified willing donor with full HLA match;
- Participants with severe iron overload at the time of screening;
- Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody;
- Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.);
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- Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the clinical investigator;
- Subjects positive with the following etiological tests: human immunodeficiency virus(HIV-1-2),human cytomegalovirus (HCMV-DNA),EB virus（EBV-DNA）,HBV (HBsAg/HBV-DNA positive),HCV antibody (HCV-Ab), Human T-lymphotropic virus antibody (HTLV-Ab), Treponema pallidum antibody (TP-Ab);
- Uncorrectable coagulation dysfunction or history of severe bleeding disorder;
- History of major organ damage including:
- Liver function test suggest AST or ALT levels >3× upper limit of normal（ULN）;
- Total serum bilirubin value>2.5×ULN;if combined with Gilbert syndrome, total bilirubin>3×ULN and direct bilirubin value>2.5×ULN;
- Left ventricular ejection fraction <45%;
- Baseline calculated eGFR<60mL/min/1.73m2;
- Pulmonary function:FEV1/FVC<60% and/or diffusion capacity of carbon monoxide (DLco) <60% of prediction;
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 2 recruiting
- Recruiting
Ruijin Hospital, Shanghai Jiaotong University School of Medicine
Shanghai, Shanghai Municipality
Wei Tang, PhD, +86 13472889588
Saijuan Chen, PhD
- Recruiting
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality
Zhen Gao, Master, +86 15522360862, Gaozhen@ihcams.ac.cn
Jun Shi, PhD
About this study
Official title: A Phase I/II Clinical Study Evaluating the Safety and Efficacy of KL003 Cell Injection in Transfusion-dependent β-thalassemia
This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with β-thalassemia major.
Read the detailed description
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess KL003 Cell Injection in up to 41 participants with transfusion-dependent β-thalassemia (TDT) who are ≥3 and ≤35 years of age. KL003 Cell Injection is autologous CD34+ stem cells transduced Ex Vivo with a lentiviral Vector encoding βA-T87Q-Globin.
- Conditions
- Transfusion-dependent Beta-Thalassemia
- Being tested
- KL003 Cell Injection Drug Product
- Keywords
- gene therapy, lentiviral vector, KL003 cell injection, transfusion-dependent β-thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KL003 Cell Injection Drug ProductDrug
Administered by intravenous infusion after myeloablative conditioning with busulfan.
| Study group | Role | What they receive |
|---|---|---|
| KL003 Cell Injection Drug Product | Experimental | Transplant of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene Receives: Drug: KL003 Cell Injection Drug Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
KL003 engraftment
Proportion of participants with successful engraftment within 42 days after KL003 infusion.
Measured: From time of KL003 infusion through Month 2
Engraftment time of neutrophil and platelet
Neutrophil engraftment was defined as the first day when neutrophils ≥ 0.5×10^9/L for 3 consecutive days; Platelet engraftment was defined as the first the first day of platelet count ≥ 20.0×10^9/L for 7 consecutive days with no platelet transfusions.
Measured: From time of KL003 infusion through Month 24
Overall Survival
Overall survival was defined as time from date of KL003 infusion to date of death.
Measured: From time of KL003 infusion through Month 24
The number, frequency and severity of adverse events (AE) within 1 year after infusion of KL003 drug products
Frequency and severity of AEs \& SAEs identified according to NCI CTCAE 5.0
Measured: From time of KL003 infusion through Month 24
Clonal dominance or secondary tumors caused by lentiviral vector insertional-mutation
Clonal dominance was defined as an ISA result greater than 90% of the total insertion sites (IS) at any time
Measured: From time of KL003 infusion through Month 24
Numbers of Participants With Vector-Derived Replication-Competent Lentivirus (RCL)
Peripheral blood samples were analyzed for detection of RCL
Measured: From time of KL003 infusion through Month 24
Secondary outcomes (4)
The proportion of participants achieved Transfusion Independence (TI)for at least 6 months
TI 6 is defined as Hb ≥ 90.0 g/L after reinfusion and without disease-related routine blood transfusion for 6 months
Measured: From time of KL003 infusion through Month 24
The proportion of participants achieved TI 12
TI 12 is defined as Hb ≥ 90.0 g/L after reinfusion and without disease-related routine blood transfusion for 12 months
Measured: From time of KL003 infusion through Month 24
The start time of Transfusion Independence (TI) after KL003 infusion
The TI start time is defined as the first day of treated participants with transfusion-dependent β-thalassemia (TDT) who achieved transfusion independence.
Measured: From time of KL003 infusion through Month 24
Total Hb and the vector-derived HbA^T87Q
The total Hb is measured by routine blood test, Therapeutic globin expression was measured by HbA^T87Q in peripheral blood.
Measured: From time of KL003 infusion through Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2024
- Study start5 Apr 2024
- Primary completion20 Nov 2027 (estimated)
- Study completion20 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transfusion-dependent Beta-ThalassemiaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 41 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kanglin Biotechnology (Hangzhou) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Haoquan Wu, PhD, Study director, R&D Kanglin Biotech
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06280378
- Sponsor's protocol ID
- CP-KL003-003/01
- Status last verified
- Oct 2026
- First submitted
- 20 Feb 2024
- Last updated
- 2 Nov 2026
- Results posted
- No
- Data monitoring committee
- 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 2 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Clinical Study Evaluating the Safety and Efficacy of KL003 Cell Injection in Transfusion-dependent β-thalassemia
- ClinicalTrials.gov ID
- NCT06280378
- Acronym / sponsor ID
- CP-KL003-003/01
- Conditions
- Transfusion-dependent Beta-Thalassemia
- Keywords
- gene therapy, lentiviral vector, KL003 cell injection, transfusion-dependent β-thalassemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 41 participants (estimated)
- Lead sponsor
- Kanglin Biotechnology (Hangzhou) Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 5 Apr 2024 (actual)
- Primary completion
- 20 Nov 2027 (estimated)
- Study completion
- 20 Dec 2028 (estimated)
- First posted
- 28 Feb 2024 (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 non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with β-thalassemia major.
Detailed description
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess KL003 Cell Injection in up to 41 participants with transfusion-dependent β-thalassemia (TDT) who are ≥3 and ≤35 years of age. KL003 Cell Injection is autologous CD34+ stem cells transduced Ex Vivo with a lentiviral Vector encoding βA-T87Q-Globin.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| KL003 Cell Injection Drug Product | Experimental | Drug: KL003 Cell Injection Drug ProductTransplant of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene |
Who may qualify
- Ages
- 3 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Male or female age between 3-35 years;
- Diagnosis of transfusion-dependent β-thalassemia and a history of at least 100 mL/kg/year of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years;
- Karnofsky performance status ≥70 for participants≥16 years of age; Lansky performance status of ≥70 for participants<16 years of age;
- Eligible to undergo auto-HSCT;
- Willing and able to follow the research procedures and conditions, with good compliance;
Key exclusion criteria (15 listed)
- Diagnosis of composite α thalassemia;
- Prior receipt of gene therapy or allo-HSCT;
- Meet the criteria for allo-HSCT and with an identified willing donor with full HLA match;
- Participants with severe iron overload at the time of screening;
- Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody;
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 |
|---|---|---|
| KL003 Cell Injection Drug ProductAdministered by intravenous infusion after myeloablative conditioning with busulfan. | Drug | KL003 Cell Injection Drug Product |
Primary outcome
KL003 engraftment
Time frame: From time of KL003 infusion through Month 2
Proportion of participants with successful engraftment within 42 days after KL003 infusion.
Engraftment time of neutrophil and platelet
Time frame: From time of KL003 infusion through Month 24
Neutrophil engraftment was defined as the first day when neutrophils ≥ 0.5×10^9/L for 3 consecutive days; Platelet engraftment was defined as the first the first day of platelet count ≥ 20.0×10^9/L for 7 consecutive days with no platelet transfusions.
Overall Survival
Time frame: From time of KL003 infusion through Month 24
Overall survival was defined as time from date of KL003 infusion to date of death.
The number, frequency and severity of adverse events (AE) within 1 year after infusion of KL003 drug products
Time frame: From time of KL003 infusion through Month 24
Frequency and severity of AEs \& SAEs identified according to NCI CTCAE 5.0
Clonal dominance or secondary tumors caused by lentiviral vector insertional-mutation
Time frame: From time of KL003 infusion through Month 24
Clonal dominance was defined as an ISA result greater than 90% of the total insertion sites (IS) at any time
Numbers of Participants With Vector-Derived Replication-Competent Lentivirus (RCL)
Time frame: From time of KL003 infusion through Month 24
Peripheral blood samples were analyzed for detection of RCL
4 secondary outcomes
The proportion of participants achieved Transfusion Independence (TI)for at least 6 months
Time frame: From time of KL003 infusion through Month 24
The proportion of participants achieved TI 12
Time frame: From time of KL003 infusion through Month 24
The start time of Transfusion Independence (TI) after KL003 infusion
Time frame: From time of KL003 infusion through Month 24
Total Hb and the vector-derived HbA^T87Q
Time frame: From time of KL003 infusion through Month 24
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 2 Nov 2026.
- Individual participant dataSharing planned: No
Related trials for Transfusion-dependent Beta-Thalassemia
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
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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.
Haoquan Wu, PhD study director
R&D Kanglin Biotech
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing KL003 Cell Injection Drug Product in people with Transfusion-dependent Beta-Thalassemia, run by Kanglin Biotechnology (Hangzhou) Co., Ltd. at 2 sites 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 7 inclusion and 15 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 2 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 jingfeng Yan (+86 18852138866, yanjingfeng@kanglinbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
KL003 engraftment (time frame: From time of KL003 infusion through Month 2). The study lists 6 primary outcomes in all. 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 2 Nov 2026; AzTrials last refreshed its copy on 6 Nov 2026.