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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-05-11

Study Completion Date

2027-08-01

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This is an interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem and Progenitor Cells (HSPCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in patients with transfusion-dependent beta-thalassemia.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector

NCT03351829

Beta-Thalassemia

UNKNOWN NA

A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease

NCT02151526

Beta-Thalassemia Major Sickle Cell Disease

COMPLETED PHASE1/PHASE2

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

NCT06271512

Beta-Thalassemia

RECRUITING

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

NCT03207009

Beta-Thalassemia

COMPLETED PHASE3

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

NCT02633943

Transfusion-dependent Beta-Thalassemia

ACTIVE_NOT_RECRUITING

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

The participant's autologous HSPCs will be transduced with the self-inactivating lentiviral vector, carrying the functional HBB gene.

Study duration per participant is approximately 27 months including an approximately 30-day screening/baseline period, an approximately 60-day mobilization and product manufacture, an approximately 10-day myeloablative conditioning, 1 treatment day, and an approximately 24-month study observation period.

The endpoints will be used to assess the safety and efficacy profiles in patients with transfusion-dependent beta-thalassemia.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Beta-Thalassemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Vebeglogene autotemcel

One-time infusion of≥5×10\^6/kg beta-globin lentiviral vector transduced HSPCs

Group Type EXPERIMENTAL

Vebeglogene autotemcel

Intervention Type DRUG

Autologous HSPCs transduced with self-inactivating lentiviral vector encoding functional HBB gene and resuspended in cryopreservative solution in the final immediate container for the intended medical use.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Vebeglogene autotemcel

Autologous HSPCs transduced with self-inactivating lentiviral vector encoding functional HBB gene and resuspended in cryopreservative solution in the final immediate container for the intended medical use.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Beta-globin lentiviral vector transduced autologous HSPCs

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No