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

NCT06219239 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2025-08-05

No results posted yet for this study

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.

Conditions

Interventions

GENETIC

KL003 cell injection Drug Product

No conditioning regimen for transfusion of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene

Sponsors & Collaborators

  • Kanglin Biotech

    collaborator UNKNOWN

  • Institute of Hematology & Blood Diseases Hospital, China

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-01-04
Primary Completion
2025-12-31
Completion
2026-12-31

Countries

  • China

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06219239 on ClinicalTrials.gov