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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
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