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Gene Therapy Trial Report

Summary

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)


NCTID NCT04443907 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Sickle Cell Disease
Disease Ontology Term DOID:0081445
Compound Name OTQ923
Sponsor Novartis Pharmaceuticals
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 4 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant HBG1/HBG2
Therapy Type Gene editing
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 Electroporation
Editor Type Cas9 RNP
Dose 1 Minimum dose: 3E6 CD34+ cells/kg
Dose 2 Dose range: 2.8-5.99E6 CD34+ cells/kg

Study Record Dates


Current Stage Phase1
Submit Date 2020-04-29
Completion Date 2025-01-06
Last Update 2026-01-08

Participation Criteria


Eligible Age 2 Years - 40 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Male or female subjects age 2-40 years inclusive 2. Confirmed diagnosis of sickle cell disease with globin typing (e.g. HbSS, HbSC, HbS/β0-thalassemia or others) 3. Performance status \>70% (Karnofsky for subjects \>16 years of age and Lansky for subjects \<16 years of age) 4. At least one of the following indicators of disease severity as defined in the protocol - Vaso-occlusive pain crisis, Acute chest syndrome, Recurrent priapism, prior stroke, receive chronic transfusions, Red cell alloimmunization 5. Subjects, who have failed, not tolerated or refused hydroxyurea therapy. Exclusion Criteria: 1. Available matched related donor for HSCT 2. Clinically significant active infection 3. Seropositive for HIV or HTLV 4. Active known malignancy, myelodysplasia, abnormal cytogenetics or immunodeficiency 5. Prior HSCT or gene therapy 6. Known hepatic cirrhosis, bridging hepatic fibrosis or active hepatitis 7. Protocol defined iron overload 8. Cerebrovascular procedure within one year, including pial synangiosis for Moyamoya 9. Severe or progressive arteriopathy or cerebrovascular disease, including Moyamoya Other protocol defined inclusion/exclusion criteria may apply
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 3
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Product was developed in collaboration with Intellia, Novartis opted to discontinue development in February 2023

Resources/Links