💬In plain words
What the trial is, and whether it could suit you
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.
Sign in to get the eligibility rules as simple questions to take to your doctor.
📋 Eligibility rules (as published)
Inclusion Criteria:
* 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 Criteria:
* 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:
1. Liver function test suggest AST or ALT levels \>3× upper limit of normal（ULN）;
2. Total serum bilirubin value\>2.5×ULN;if combined with Gilbert syndrome, total bilirubin\>3×ULN and direct bilirubin value\>2.5×ULN;
3. Left ventricular ejection fraction \<45%;
4. Baseline calculated eGFR\<60mL/min/1.73m2;
5. Pulmonary function:FEV1/FVC\<60% and/or diffusion capacity of carbon monoxide (DLco) \<60% of prediction;
💊
What it tests
- KL003 Cell Injection Drug Product
Ruijin Hospital, Shanghai Jiaotong University School of Medicine
Shanghai, China
Institute of Hematology & Blood Diseases Hospital
Tianjin, China
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
HealthTeky