Transfusion-dependent Beta-ThalassemiaNCT06219239
Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients
A study testing KL003 cell injection Drug Product in people with Transfusion-dependent Beta-Thalassemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells transfusion in subjects with transfusion-dependent β-thalassemia. 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
- 3 participants
- Where
- 1 siteChina
- Study dates
- 4 Jan 2024to 31 Dec 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
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
- Documented baseline, or pretransfusion, Hb level≤7 g/dL
- Karnofsky performance status ≥70 for subjects≥16 years of age; Lansky performance status of ≥70 for subjects<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 and maintain detailed medical records, including transfusion history
- Subject and/or legal guardians voluntarily participated in this clinical trial and signed the informed consent form, and can complete all follow-ups in accordance with the protocol requirements
Who cannot join
- Presence of clear contraindications for hematopoietic stem cell collection
- Diagnosis of composite α thalassemia
- A white blood cell (WBC) count <3×10^9/L, and/or platelet count <100×10^9/L not related to hypersplenism
- Subjects with severe iron overload at the time of screening: severe iron overload of the liver showed by MRI, serum ferritin ≥ 5000 ng/mL, or moderate to severe iron overload of the heart
- Any prior or current malignancy or myeloproliferative or significant immunodeficiency disorder
- Meet the criteria for allo-HSCT and with an identified willing donor with a full HLA match
Show all 32Show fewer
- Prior receipt of gene therapy or allo-HSCT
- Subjects with any severe active fungal, bacterial, viral, tuberculosis or other infection, including active hepatitis B (defined as serum HBV-DNA ≥2000 IU/ml), active hepatitis C virus, HCV) infection, human immunodeficiency virus (HIV) antibody-positive or active syphilis patients, etc.
- Immediate family member (i.e. parent or siblings) with a known Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome and familial adenomatous polyposis)
- Diagnosis of a significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study
- 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;
- History of bridging fibrosis, cirrhosis;
- Left ventricular ejection fraction <45%;
- New York Heart Association (NYHA) class III or IV congestive heart failure;
- Severe arrhythmia requiring medical treatment;
- Uncontrolled hypertension or unstable angina pectoris;
- Myocardial infarction or bypass or stent surgery within 12 months before drug administration;
- Valvular disease with clinical significance;
- Baseline calculated eGFR<60mL/min/1.73m2;
- Pulmonary function: FEV1/FVC<60% and/or diffusion capacity of carbon monoxide (DLco) <60% of prediction;
- Evidence of clinically significant pulmonary hypertension requiring medical intervention.
- Uncorrectable coagulation dysfunction or history of severe bleeding disorder
- Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician
- Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.)
- Participated in other clinical studies within 3 months prior to screening
- Inoculated live vaccine within 6 weeks prior to screening
- Pregnancy or breastfeeding women; Subjects or their sexual partners were unable to take medically recognized effective contraceptive measures during the 27-month study period
- The subjects or their parents would not comply with the study procedures outlined in the protocol
- The subjects received hydroxyurea or thalidomide or hypomethylating drugs within 3 months before hematopoietic stem cell collection
- Patients considered to be ineligible for the study by the investigator for reasons other than the above
Trial locations and contacts
1 site in 1 country.
China1 site
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
About this study
Official title: Evaluation of the Safety and Efficacy of KL003 Gene Therapy in Patients With Transfusion-dependent β-thalassemia With No Conditioning Regimen
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells transfusion in subjects with transfusion-dependent β-thalassemia.
- Conditions
- Transfusion-dependent Beta-Thalassemia
- Being tested
- KL003 cell injection Drug Product
- Keywords
- Beta-Thalassemia, Hematopoietic Stem-Cell Transplantation, KL003, Gene therapy, no conditioning regimen
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KL003 cell injection Drug ProductGenetic
No conditioning regimen for transfusion of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene
| Study group | Role | What they receive |
|---|---|---|
| KL003 cell injection Drug Product | Experimental | Traditional myeloablative conditioning regimen consists of Busulfan, which may increase the risk of irreversible pulmonary fibrosis, VOD, and infertility due to potential serious toxicity. In this study, we intend to use genetic hematopoietic stem cells (lentivirus transduction) transfusion with no conditioning regimen, which could avoid toxicity due to chemotherapy drugs. Receives: Genetic: KL003 cell injection Drug Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number, frequency and severity of adverse events (AE) after reinfusion of KL003 drug products
Measured: within 6 months
The proportion of participants who meet the definition of transfusion independence (TI) for at least 6 months
TI is defined as Hb ≥ 90.0 g/L after reinfusion and without disease-related routine blood transfusion for 6 months
Measured: Up to 24 months post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2024
- Study start4 Jan 2024
- Primary completion31 Dec 2025
- Study completion31 Dec 2025
Why it stopped: Suboptimal treatment response
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.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Kanglin Biotech
- Responsible party
- Principal investigator, Jun Shi, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06219239
- Sponsor's protocol ID
- IIT2023084
- Status last verified
- Nov 2026
- First submitted
- 5 Jan 2024
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Safety and Efficacy of KL003 Gene Therapy in Patients With Transfusion-dependent β-thalassemia With No Conditioning Regimen
- ClinicalTrials.gov ID
- NCT06219239
- Acronym / sponsor ID
- IIT2023084
- Conditions
- Transfusion-dependent Beta-Thalassemia
- Keywords
- Beta-Thalassemia, Hematopoietic Stem-Cell Transplantation, KL003, Gene therapy, no conditioning regimen
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Suboptimal treatment response
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Kanglin Biotech
- Study start
- 4 Jan 2024 (actual)
- Primary completion
- 31 Dec 2025 (actual)
- Study completion
- 31 Dec 2025 (actual)
- First posted
- 23 Jan 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. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells transfusion in subjects with transfusion-dependent β-thalassemia.
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 | Genetic: KL003 cell injection Drug ProductTraditional myeloablative conditioning regimen consists of Busulfan, which may increase the risk of irreversible pulmonary fibrosis, VOD, and infertility due to potential serious toxicity. In this study, we intend to use genetic hematopoietic stem cells (lentivirus transduction) transfusion with no conditioning regimen, which could avoid toxicity due to chemotherapy drugs. |
Who may qualify
- Ages
- 3 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 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
- Documented baseline, or pretransfusion, Hb level≤7 g/dL
- Karnofsky performance status ≥70 for subjects≥16 years of age; Lansky performance status of ≥70 for subjects<16 years of age
- Eligible to undergo auto-HSCT
Key exclusion criteria (32 listed)
- Presence of clear contraindications for hematopoietic stem cell collection
- Diagnosis of composite α thalassemia
- A white blood cell (WBC) count <3×10^9/L, and/or platelet count <100×10^9/L not related to hypersplenism
- Subjects with severe iron overload at the time of screening: severe iron overload of the liver showed by MRI, serum ferritin ≥ 5000 ng/mL, or moderate to severe iron overload of the heart
- Any prior or current malignancy or myeloproliferative or significant immunodeficiency disorder
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 ProductNo conditioning regimen for transfusion of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene | Genetic | KL003 cell injection Drug Product |
Primary outcome
The number, frequency and severity of adverse events (AE) after reinfusion of KL003 drug products
Time frame: within 6 months
The proportion of participants who meet the definition of transfusion independence (TI) for at least 6 months
Time frame: Up to 24 months post transplant
TI is defined as Hb ≥ 90.0 g/L after reinfusion and without disease-related routine blood transfusion for 6 months
Results and safety
The study was stopped early (reason given: Suboptimal treatment response). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2026.
Related trials for Transfusion-dependent Beta-Thalassemia
Active, not recruiting
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
Transfusion-dependent Beta-Thalassemia
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
RecruitingPhase 3
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing KL003 cell injection Drug Product in people with Transfusion-dependent Beta-Thalassemia, run by Institute of Hematology & Blood Diseases Hospital, China 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 8 inclusion and 32 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: stopped early, as of the sponsor's last update on 4 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 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.
Are results available for this study?
The study was stopped early (reason given: Suboptimal treatment response). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The number, frequency and severity of adverse events (AE) after reinfusion of KL003 drug products (time frame: within 6 months). The study lists 2 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 4 Nov 2026.