Ignite Creation Date:
2025-12-24 @ 2:41 PM
Ignite Modification Date:
2025-12-26 @ 7:06 AM
Study NCT ID:
NCT06308159
Status:
RECRUITING
Last Update Posted:
2025-11-25
First Post:
2024-03-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
Sponsor:
Lantu Biopharma
Organization:
Study Overview
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
Status:
RECRUITING
Status Verified Date:
2025-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: