Clinical trial NCT06308159: An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
Official title: An Open-label Clinical Trial of Ex Vivo Beta-globin Lentiviral Vector Transduction of Autologous CD34+HSPCs (Vebeglogene Autotemcel) for the Treatment of Transfusion Dependent Beta-thalassemia Patients
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Beta-Thalassemia
Lead sponsor: Lantu Biopharma
Sponsor class: INDUSTRY
First posted: 2024-03-13
Enrollment: 6
Sex: ALL
Maximum age (years): 35
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

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.

Primary outcomes:
measure: Time and duration of the subject's hemoglobin (Hb)≥9.0 g/dL without receiving red blood cell infusion
timeFrame: From baseline to Month 24
Secondary outcomes:
measure: The prevalence and severity of adverse events (AEs) and serious adverse events (SAEs)
timeFrame: From baseline to Month 24
measure: The reduction of red blood cells (RBCs) transfusion requirement after product infusion compared to previous transfusion records
timeFrame: From infusion to Month 24
measure: Number of days required to achieve successful neutrophil and platelet engraftment
timeFrame: From infusion to Month 24
measure: Vector copy number (VCN) in peripheral blood over time
timeFrame: From baseline to Month 24
Other outcomes:
Locations:
city: Kunming
country: China
facility: 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
state: Yunnan
city: Kunming
country: China
facility: Kunming Hope of Health Hospital
state: Yunnan
Study officials:
References: