Transfusion-dependent Beta-ThalassemiaNCT05860595
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
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 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
- 23 May 2023to 24 Oct 2028
- Phase
- Interventional
- 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
- 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-up in accordance with the protocol requirements
Who cannot join
- 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), Treponema pallidum antibody (TP-Ab)
- Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the clinical investigator
- Contraindication to bone marrow collection
- Any prior or current malignancy or myeloproliferative or significant immunodeficiency disorder
- A white blood cell (WBC) count <3×10^9/L, and/or platelet count <100×10^9/L not related to hypersplenism
- Diagnosis of composite α thalassemia
Show all 34Show fewer
- Participants 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
- Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody
- Meet the criteria for allo-HSCT and with an identified willing donor with a full HLA match
- Prior receipt of gene therapy or allo-HSCT
- 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.)
- Participation in another clinical study with an investigational drug within 30 days of Screening or participating in another clinical study with an investigational drug
- 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
- Receipt of hydroxyurea therapy within 3 months before HSCT harvest
- 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.
About this study
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 in subjects with transfusion-dependent β-thalassemia.
Read the detailed description
Subject participation for this study will be 24 months. Subjects who enroll in this study will be asked to participate in a subsequent 13-year follow-up for gene therapy products.
- Conditions
- Transfusion-dependent Beta-Thalassemia
- Being tested
- KL003 cell injection Drug Product
- Keywords
- Beta-Thalassaemia, Hematopoietic Stem-Cell Transplantation
- 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
Transplant of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene.
| Study group | Role | What they receive |
|---|---|---|
| KL003 cell injection Drug Product | Experimental | Each recruited subject will accept KL003 Transplantation. Receives: Genetic: KL003 cell injection Drug Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of participants with successful lentiviral vector transduced CD34+ stem cell engraftment
Successful engraftment was defined as neutrophil count [ANC] ≥0.5×10^9/L for 3 consecutive days
Measured: Up to 42 days post transplant
Engraftment time of neutrophil
The first day when neutrophils ≥ 0.5×10^9/L for 3 consecutive days
Measured: Up to 42 days post transplant
Engraftment time of platelet
The first day of platelet count ≥ 20.0×10^9/L for 7 consecutive days after platelet transfusion independence
Measured: Up to 42 days post transplant
Transplant-related mortality within 100 days and within 1 year after reinfusion of KL003 drug product
Measured: Up to 1 year post transplant
The number, frequency and severity of adverse events (AE) within 1 year after reinfusion of KL003 drug products
Frequency and severity of AEs \& SAEs identified according to NCI CTCAE 5.0
Measured: Up to 24 months post transplant
Secondary outcomes (3)
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
The duration of transfusion independence
Measured: Up to 24 months post transplant
Changes in the frequency and volume of blood transfusion
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 posted16 May 2023
- Study start23 May 2023
- Primary completion20 Aug 2027 (estimated)
- Study completion24 Oct 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.
- 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 (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- R&D Kanglin Biotech
- Responsible party
- Principal investigator, Jun Shi, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05860595
- Sponsor's protocol ID
- IIT2023021
- Status last verified
- Nov 2026
- First submitted
- 8 May 2023
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- 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 the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
- ClinicalTrials.gov ID
- NCT05860595
- Acronym / sponsor ID
- IIT2023021
- Conditions
- Transfusion-dependent Beta-Thalassemia
- Keywords
- Beta-Thalassaemia, Hematopoietic Stem-Cell Transplantation
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- R&D Kanglin Biotech
- Study start
- 23 May 2023 (actual)
- Primary completion
- 20 Aug 2027 (estimated)
- Study completion
- 24 Oct 2028 (estimated)
- First posted
- 16 May 2023 (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 in subjects with transfusion-dependent β-thalassemia.
Detailed description
Subject participation for this study will be 24 months. Subjects who enroll in this study will be asked to participate in a subsequent 13-year follow-up for gene therapy products.
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 ProductEach recruited subject will accept KL003 Transplantation. |
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 (34 listed)
- 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), Treponema pallidum antibody (TP-Ab)
- Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the clinical investigator
- Contraindication to bone marrow collection
- Any prior or current malignancy or myeloproliferative or significant immunodeficiency disorder
- A white blood cell (WBC) count <3×10^9/L, and/or platelet count <100×10^9/L not related to hypersplenism
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 ProductTransplant of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene. | Genetic | KL003 cell injection Drug Product |
Primary outcome
Percentage of participants with successful lentiviral vector transduced CD34+ stem cell engraftment
Time frame: Up to 42 days post transplant
Successful engraftment was defined as neutrophil count [ANC] ≥0.5×10^9/L for 3 consecutive days
Engraftment time of neutrophil
Time frame: Up to 42 days post transplant
The first day when neutrophils ≥ 0.5×10^9/L for 3 consecutive days
Engraftment time of platelet
Time frame: Up to 42 days post transplant
The first day of platelet count ≥ 20.0×10^9/L for 7 consecutive days after platelet transfusion independence
Transplant-related mortality within 100 days and within 1 year after reinfusion of KL003 drug product
Time frame: Up to 1 year post transplant
The number, frequency and severity of adverse events (AE) within 1 year after reinfusion of KL003 drug products
Time frame: Up to 24 months post transplant
Frequency and severity of AEs \& SAEs identified according to NCI CTCAE 5.0
3 secondary outcomes
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
The duration of transfusion independence
Time frame: Up to 24 months post transplant
Changes in the frequency and volume of blood transfusion
Time frame: Up to 24 months post transplant
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 4 Nov 2026.
Related trials for Transfusion-dependent Beta-Thalassemia
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β-Thalassemia Treatment With KL003 Cell Injection
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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
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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)
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ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
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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. 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 34 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: under way, not looking for participants, 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?
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?
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?
Percentage of participants with successful lentiviral vector transduced CD34+ stem cell engraftment (time frame: Up to 42 days post transplant). The study lists 5 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.