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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome
NCTID
NCT03837483
(View at clinicaltrials.gov)
Description
This is an open-label, single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy. OTL-103 consists of autologous CD34+ hematopoietic stem cells in which the gene encoding for the Wiskott-Aldrich Syndrome is introduced by means of a third generation lentiviral vector.
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Development Status
Active
Indication
Wiskott-Aldrich Syndrome
Disease Ontology Term
DOID:9169
Compound Name
Etuvetidigene autotemcel
Compound Alias
TLT003, OTL-103
Sponsor
Fondazione Telethon
Funder Type
Other
Recruitment Status
Active not recruiting
Enrollment Count
10
Results Posted
Not Available
Therapy Information
Target Gene/Variant
WAS
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 (4.4 - 14.5E6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase3
Submit Date
2019-02-08
Completion Date
2027-09
Last Update
2024-01-31
Participation Criteria
Eligible Age
<=65 Years
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
2
Locations
United States,Italy
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
BLA submitted 3/11/25
Resources/Links
Clinical Publications
Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy
(Abstract) Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010 - ASH 2019
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study
B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome
News and Press Releases
Fondazione Telethon submits US marketing authorization application for etuvetidigene autotemcel gene therapy for the treatment of Wiskott-Aldrich syndrome
Inside the efforts to rescue a rare disease gene therapy
Preclinical Publications
Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction
Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome
Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models
Related NCTID
Phase 1/2: NCT01347242 (same vector, sponsored by Genethon)
Phase 1/2: NCT01410825
Phase 1/2: NCT01347346 (same vector, sponsored by Genethon)
Phase 1/2: NCT01515462
LTFU: NCT02333760 (sponsored by Genethon)