RecruitingNot ApplicableNCT06219239

Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients

Evaluation of the Safety and Efficacy of KL003 Gene Therapy in Patients With Transfusion-dependent β-thalassemia With No Conditioning Regimen

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Enrollment

3 participants

Start Date

Jan 4, 2024

Study Type

INTERVENTIONAL

Conditions

Transfusion Dependent Beta-Thalassemia

Summary

This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells transfusion in subjects with transfusion-dependent β-thalassemia.

Eligibility

Min Age: 3 YearsMax Age: 35 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying KL003 cell injection Drug Product for people with transfusion dependent beta-thalassemia. The study is currently recruiting participants at 1 location. People eligible for this study include aged 3 Years to 35 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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