Clinical trial
A Phase I/II Clinical Study Evaluating the Safety and Efficacy of KL003 Cell Injection in Transfusion-dependent β-thalassemia
Name
CP-KL003-003/01
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.
Trial arms
Trial start
2024-02-01
Estimated PCD
2025-05-01
Trial end
2027-05-01
Status
Not yet recruiting
Phase
Early phase I
Treatment
KL003 Cell Injection Drug Product
Administered by intravenous infusion after myeloablative conditioning with busulfan.
Arms:
KL003 Cell Injection Drug Product
Size
41
Primary endpoint
KL003 engraftment
From time of KL003 infusion through Month 2
Engraftment time of neutrophil and platelet
From time of KL003 infusion through Month 24
Overall Survival
From time of KL003 infusion through Month 24
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
Clonal dominance or secondary tumors caused by lentiviral vector insertional-mutation
From time of KL003 infusion through Month 24
Numbers of Participants With Vector-Derived Replication-Competent Lentivirus (RCL)
From time of KL003 infusion through Month 24
Eligibility criteria
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;
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 41, 'type': 'ESTIMATED'}}
Updated at
2024-02-28
1 organization
1 product
1 indication
Product
KL003 Cell InjectionIndication
Beta-ThalassemiaOrganization
Kanglin Biotechnology (Hangzhou)