Β-Thalassemia Treatment with KL003 Cell Injection

Recruiting now · Phase 1/Phase 2

Conditions studied: Transfusion-dependent Beta-Thalassemia

In brief

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.

Key facts

Study ID

NCT06280378

Run by

Kanglin Biotechnology (Hangzhou) Co., Ltd.

People needed

41

Starts

2024-04-05

Expected to finish

2027-05-01

Last updated by the study team

2025-03-10

Who can join

Age: 3 and older, up to 35. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.);
• 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;

Where it is running

• Ruijin Hospital, Shanghai Jiaotong University School of Medicine — Shanghai, Shanghai Municipality, China (enrolling)
• Institute of Hematology & Blood Diseases Hospital — Tianjin, Tianjin Municipality, China (enrolling)

Full record on ClinicalTrials.gov

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