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

Plain-language summary

This study evaluates Vebeglogene autotemcel, a gene therapy, in patients with transfusion-dependent beta-thalassemia. It enrolls patients with a diagnosis of beta-thalassemia and a history of red blood cell transfusions who have low hemoglobin levels. The study is in Phase 1/Phase 2 and is active but not currently recruiting.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Active, not recruiting

Sponsor

Lantu Biopharma

Intervention

Vebeglogene autotemcel

Start date

5/11/2024

Estimated completion

8/1/2027

Countries

China

Related genes

Locations

• Kunming, China
• Kunming, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.