Β-Thalassemia Treatment with KL003 Cell Injection
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.
| Condition(s) | Transfusion-dependent Beta-Thalassemia |
|---|---|
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Study type | Interventional |
| 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. |
| Who can participate | 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 a |
| Ages | 3 Years to 35 Years |
| Sex | All |
| Lead sponsor | Kanglin Biotechnology (Hangzhou) Co., Ltd. |
| Locations | Shanghai, Shanghai Municipality, China; Tianjin, Tianjin Municipality, China |
| Start date | 2024-04-05 |
| NCT ID | NCT06280378 |
| Official listing | https://clinicaltrials.gov/study/NCT06280378 |