A Phase I/II Clinical Study Evaluating the Safety and Efficacy of KL003 Cell Injection in Transfusion-dependent Β-thalassemia

NCT06280378 Phase 1/Phase 2 RECRUITING

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.

Details

Lead sponsor	Kanglin Biotechnology (Hangzhou) Co., Ltd.
Phase	Phase 1/Phase 2
Status	RECRUITING
Enrolment	41
Start date	2024-04-05
Completion	2027-05

Conditions

• Transfusion-dependent Beta-Thalassemia

Interventions

• KL003 Cell Injection Drug Product

Primary outcomes

• 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

Countries

China