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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Stem Cell Gene Therapy for Sickle Cell Disease
NCTID
NCT02247843
(View at clinicaltrials.gov)
Description
This Phase I clinical trial will assess the safety and initial evidence for efficacy of an autologous transplant of lentiviral vector modified peripheral blood for adults with severe sickle cell disease.
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Indication
Sickle Cell Disease
Compound Name
Lenti/G-βAS3-FB (contains anti-sickling mutations: T87Q, G16D, E22A)
Sponsor
Donald B. Kohn, M.D.
Funder Type
Other
Status
Active not recruiting
Enrollment Count
4
Therapy Information
Target Gene/Variant
HBB
Therapy Type
Gene transfer
Therapy Route
Ex-vivo
Mechanism of Action
Overexpression of protective allele/gene
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
2014-09-20
Completion Date
2025-12
Last Update
2024-05-16
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Eligible Sex
ALL
Locations
No.of Trial Sites
1
Locations
United States
Regulatory Information
Has US IND
True
Recent Updates
Uses the same βAS3-globin as NCT03964792
Resources/Links
Clinical Publications
β-globin gene transfer to human bone marrow for sickle cell disease
Preclinical Publications
β-globin gene transfer to human bone marrow for sickle cell disease
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease
Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study
β-Globin Lentiviral Vectors Have Reduced Titers due to Incomplete Vector RNA Genomes and Lowered Virion Production
Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells