NCT06280378RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
A Phase I/II Clinical Study Evaluating the Safety and Efficacy of KL003 Cell Injection in Transfusion-dependent β-thalassemia
Source: ClinicalTrials.gov · Last updated 2 Nov 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 41 (estimated)
- First submitted
- 20 Feb 2024
- Start date
- 5 Apr 2024
- Primary completion date
- 20 Nov 2027
- Lead sponsor
- Kanglin Biotechnology (Hangzhou) Co., Ltd.
- Diseases
- Thalassaemia
- NCT number
- NCT06280378
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 41
- From 5 Apr 2024 to the primary completion date, 20 Nov 2027
- 2 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior receipt of gene therapy or allo-HSCT;
Other illness
- 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;
Lab values
- 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;
Other
- 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;
Who the study is looking for
- 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.
Age in the registry: 3 years to 35 years · Sex: All · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Ruijin Hospital Shanghai Jiaotong University School of Medicine Coordinator of this site? Confirm its status | Shanghai | China | Recruiting |
| Institute of Hematology & Blood Diseases Hospital Coordinator of this site? Confirm its status | Tianjin | China | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.