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Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Therapy for Transfusion Dependent Beta-thalassemia
NCTID
NCT02453477
(View at clinicaltrials.gov)
Description
This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia
(Show More)
Development Status
Inactive
Indication
Beta-Thalassemia Major
Disease Ontology Term
DOID:0080771
Compound Name
OTL-300
Sponsor
IRCCS San Raffaele
Funder Type
Other
Recruitment Status
Unknown
Enrollment Count
10
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
Intraosseous
Drug Product Type
Autologous cells
Target Tissue/Cell
CD34+ cells
Delivery System
Viral transduction
Vector Type
LV
Editor Type
none
Dose 1
Target dose: 5E6 CD34+ cells/kg
Dose 2
Minimum dose: 2E6 CD34+ cells/kg
Dose 3
Maximum dose: 20E6 CD34+ cells/kg
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2015-05-15
Completion Date
2019-08
Last Update
2019-06-28
Participation Criteria
Eligible Age
3 Years - 64 Years
Standard Ages
Child, Adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
Italy
Regulatory Information
Has US IND
FDA Designations
Recent Updates
Program discontinued by Orchard
Resources/Links
Clinical Publications
Bone marrow stromal cells from β-thalassemia patients have impaired hematopoietic supportive capacity
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent β-thalassemia
(Abstract #49) Gene Therapy for the Treatment of Adult and Pediatric Patients Affected by Transfusion Dependent Beta-Thalassemia - ASGCT 2019
Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients
News and Press Releases
Orchard Therapeutics Unveils New Strategic Plan and Reports First Quarter 2020 Financial Results
Preclinical Publications
The GATA1-HS2 enhancer allows persistent and position-independent expression of a β-globin transgene
Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia
In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia