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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
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
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