Testing KL003 Cell Injection for Transfusion-Dependent Beta-Thalassemia
Tests KL003 cell injection in people with transfusion-dependent beta-thalassemia major.
Plain-language title generated from the registry record
Registry title: β-Thalassemia Treatment With KL003 Cell Injection
Key facts
- Where
- Shanghai and Tianjin, ChinaSee the 2 sites
- Phase
- Phase 1/2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 3 and 35 years old?
Source: NCT06280378, who can take part
Where in China
- Ruijin Hospital Shanghai Jiaotong University School of MedicineShanghai, ChinaTaking people
- Institute of Hematology & Blood Diseases HospitalTianjin, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Kanglin Biotechnology (Hangzhou) Co., Ltd.
- Trial code
- NCT06280378
- Record updated
- 2 November 2026
- Original description
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 full original descriptionShow less
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.
Inclusion: 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.
Exclusion: Prior receipt of gene therapy or allo-HSCT; Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.); 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; Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody; 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; Diagnosis of composite α thalassemia; 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.