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Clinical Trial Report
Gene Therapy Trial Report
Summary
Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
NCTID
NCT04418414
(View at clinicaltrials.gov)
Description
This is a first-in-human, non-randomized, open label, single treatment, Phase 1 study in approximately 7 patients with severe hemophilia A. The study will evaluate gene therapy by transplantation of autologous CD34+ hematopoietic stem cells transduced ex vivo with the CD68-ET3 lentiviral vector.
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Development Status
Active
Indication
Hemophilia A
Disease Ontology Term
DOID:12134
Compound Name
CD68-ET3
Sponsor
Expression Therapeutics, LLC
Funder Type
Industry
Recruitment Status
Not yet recruiting
Enrollment Count
7
Results Posted
Not Available
Therapy Information
Target Gene/Variant
F8
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
Submit Date
2020-05-28
Completion Date
2039-08
Last Update
2024-02-20
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
Locations
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Resources/Links
Preclinical Publications
Comparison of different gene addition strategies to modify placental derived-mesenchymal stromal cells to produce FVIII
Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A
High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells
Transplanting FVIII/ET3-secreting cells in fetal sheep increases FVIII levels long-term without inducing immunity or toxicity
Non-genotoxic conditioning facilitates hematopoietic stem cell gene therapy for hemophilia A using bioengineered factor VIII
Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A
Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery