NCT06280378

Β-Thalassemia Treatment with KL003 Cell Injection

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 41 (estimated)
• Sponsor: Kanglin Biotechnology (Hangzhou) Co., Ltd. · Industry
• Sex: All
• Age: 3 Years – 35 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess KL003 Cell Injection in up to 41 participants with transfusion-dependent β-thalassemia (TDT) who are ≥3 and ≤35 years of age. KL003 Cell Injection is autologous CD34+ stem cells transduced Ex Vivo with a lentiviral Vector encoding βA-T87Q-Globin.

Conditions

• Transfusion-dependent Beta-Thalassemia

Interventions

Type	Name	Description
DRUG	KL003 Cell Injection Drug Product	Administered by intravenous infusion after myeloablative conditioning with busulfan.

Timeline

• Start date: 2024-04-05
• Primary completion: 2025-10-01
• Completion: 2027-05-01
• First posted: 2024-02-28
• Last updated: 2025-03-10

Locations

2 sites across 1 country: China