Β-Thalassemia Treatment with KL003 Cell Injection
Recruiting
Phase 1
Phase 2
Interventional Study
Transfusion-dependent Beta-Thalassemia
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 3 – 35
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 41 (estimated)
- Sponsor
- Kanglin Biotechnology (Hangzhou) Co., Ltd. · Industry
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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.
Trial Locations
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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 p…
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