Clinical Trials in China / NCT06280378
Recruiting
Phase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
NCT06280378 · tracked via the Priya Life Science China tracker
Condition(s) studied
Transfusion-dependent Beta-Thalassemia
Investigational drug(s) / intervention(s)
KL003 Cell Injection Drug Product
KL003 Cell Injection Drug Product: Administered by intravenous infusion after myeloablative conditioning with busulfan.
Study summary
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.
Eligibility
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;
Primary outcome measure(s)
- KL003 engraftment — From time of KL003 infusion through Month 2
Proportion of participants with successful engraftment within 42 days after KL003 infusion. - Engraftment time of neutrophil and platelet — From time of KL003 infusion through Month 24
Neutrophil engraftment was defined as the first day when neutrophils ≥ 0.5×10\^9/L for 3 consecutive days; Platelet engraftment was defined as the first the first day of platelet count ≥ 20.0×10\^9/L for 7 consecutive days with no platelet transfusions. - Overall Survival — From time of KL003 infusion through Month 24
Overall survival was defined as time from date of KL003 infusion to date of death. - The number, frequency and severity of adverse events (AE) within 1 year after infusion of KL003 drug products — From time of KL003 infusion through Month 24
Frequency and severity of AEs \& SAEs identified according to NCI CTCAE 5.0 - Clonal dominance or secondary tumors caused by lentiviral vector insertional-mutation — From time of KL003 infusion through Month 24
Clonal dominance was defined as an ISA result greater than 90% of the total insertion sites (IS) at any time - Numbers of Participants With Vector-Derived Replication-Competent Lentivirus (RCL) — From time of KL003 infusion through Month 24
Peripheral blood samples were analyzed for detection of RCL
Trial sites (2)
| Facility | City | Region | Status |
|---|---|---|---|
| Ruijin Hospital, Shanghai Jiaotong University School of Medicine | Shanghai | Shanghai Municipality | Recruiting |
| Institute of Hematology & Blood Diseases Hospital | Tianjin | Tianjin Municipality | Recruiting |
Other trials for the same condition
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06280378 on ClinicalTrials.gov ↗ ← All trials in China