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Gene Therapy Trial Report

Summary

An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia


NCTID NCT06308159 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Beta-Thalassemia Major
Disease Ontology Term DOID:0080771
Compound Name Vebeglogene autotemcel
Sponsor Lantu Biopharma
Funder Type Industry
Recruitment Status
Enrollment Count 6 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant HBB
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type LV
Editor Type none
Dose 1 Minimum dose: 5.0E6 CD34+ cells/kg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-03-06
Completion Date 2027-08-01
Last Update 2025-11-25

Participation Criteria


Eligible Age <=35 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Patients or parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedules. * Diagnosis of beta-thalassemia and a history of RBCs transfusions. * Documented baseline, or pretransfusion, Hb≤7 g/dL. * Availability of an adequate and well-documented transfusion history. Exclusion Criteria: * Active bacterial, viral, fungal, or parasitic infection. * A white blood cell (WBC) counts\<3×10\^9/L, and/or platelet counts\<100×10\^9/L not related to hypersplenism. * Uncorrected bleeding disorder. * Presence of severe diseases that judged not compatible with the study procedures, such as severe hepatic disease, kidney disease, lung disease, and/or cardiovascular disease. * Uncontrolled seizure disorder. * Any evidence of severe iron overload that, in the investigator's opinion, warrants exclusion. * Prior autologous hematopoietic stem cell transplantation. * Prior receipt of gene therapy.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 2
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links


Resources/Links

No External Links Available.