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Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Editing For Sickle Cell Disease
NCTID
NCT06506461
(View at clinicaltrials.gov)
Description
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD. Secondary Objective * To assess the efficacy autologous infusion of CRISPR/Cas9 genome-edited CD34+ HSPCs into patients with severe SCD.
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Development Status
Active
Indication
Sickle Cell Disease
Disease Ontology Term
DOID:0081445
Compound Name
Gene-modified CD34+ cells
Sponsor
St. Jude Children's Research Hospital
Funder Type
Other
Recruitment Status
Recruiting
Enrollment Count
25
Results Posted
Not Available
Therapy Information
Target Gene/Variant
BCL11A
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
Vector Type
Editor Type
SpCas9 mRNA
Dose 1
Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2024-06-14
Completion Date
2032-12
Last Update
2025-03-25
Participation Criteria
Eligible Age
18 Years - 24 Years
Standard Ages
Adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Resources/Links
Preclinical Publications
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease
Related NCTID
Long Term Follow-Up: NCT06646640