Β-Thalassemia Treatment with KL003 Cell Injection
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age3-35
SponsorKanglin Biotechnology (Hangzhou) Co., Ltd.
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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;
Disqualifiers
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;
Trial design
Design model
Single group
Treatments tested in this trial
KL003 Cell Injection Drug Product
DrugAdministered by intravenous infusion after myeloablative conditioning with busulfan.
Treatment groups
41 Participants
are divided into 1 treatment groupGroup A: KL003 Cell Injection Drug ProductExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
KL003 engraftment
Proportion of participants with successful engraftment within 42 days after KL003 infusion.
Time frame
From time of KL003 infusion through Month 22
Engraftment time of neutrophil and platelet
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.
Time frame
From time of KL003 infusion through Month 243
Overall Survival
Overall survival was defined as time from date of KL003 infusion to date of death.
Time frame
From time of KL003 infusion through Month 244
The number, frequency and severity of adverse events (AE) within 1 year after infusion of KL003 drug products
Frequency and severity of AEs \& SAEs identified according to NCI CTCAE 5.0
Time frame
From time of KL003 infusion through Month 24Secondary outcomes
1
The proportion of participants achieved Transfusion Independence (TI)for at least 6 months
TI 6 is defined as Hb ≥ 90.0 g/L after reinfusion and without disease-related routine blood transfusion for 6 months
Time frame
From time of KL003 infusion through Month 242
The proportion of participants achieved TI 12
TI 12 is defined as Hb ≥ 90.0 g/L after reinfusion and without disease-related routine blood transfusion for 12 months
Time frame
From time of KL003 infusion through Month 243
The start time of Transfusion Independence (TI) after KL003 infusion
The TI start time is defined as the first day of treated participants with transfusion-dependent β-thalassemia (TDT) who achieved transfusion independence.
Time frame
From time of KL003 infusion through Month 244
Total Hb and the vector-derived HbA^T87Q
The total Hb is measured by routine blood test, Therapeutic globin expression was measured by HbA\^T87Q in peripheral blood.
Time frame
From time of KL003 infusion through Month 24Sponsors and contacts
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Source ClinicalTrials.gov