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
Qualifiers
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
Treatments tested in this trial
- KL003 Cell Injection Drug Product
Treatment groups
41 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov