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Clinical Trial Report
Gene Therapy Trial Report
Summary
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
NCTID
NCT06364774
(View at clinicaltrials.gov)
Description
The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells. This is done by collecting blood stem cells from the subject, modifying those cells, adding a healthy beta globin gene, and then giving them back to the subject. It is hoped that these modified cells will decrease the need for blood transfusions. The gene modified blood stem cells are called CHOP-ALS20 ("study drug"). This experimental gene therapy has not been tried on human beings before and is not FDA approved.
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Development Status
Active
Indication
Beta-Thalassemia Major
Disease Ontology Term
DOID:0080771
Compound Name
CHOP-ALS20
Compound Description
βA-T87Q-globin
Sponsor
Children's Hospital of Philadelphia
Funder Type
Other
Recruitment Status
Recruiting
(Click here for study contact information)
Enrollment Count
12
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
Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2024-03-26
Completion Date
2027-12-31
Last Update
2025-04-23
Participation Criteria
Eligible Age
18 Years - 35 Years
Standard Ages
Adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Resources/Links
Preclinical Publications
Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies