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Clinical Trial Report
Gene Therapy Trial Report
Summary
Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
NCTID
NCT06325709
(View at clinicaltrials.gov)
Description
Background: Chronic granulomatous disease (CGD) is a rare immune disorder caused by a mutation in the CYBB gene. People with CGD have white blood cells that do not work properly. This places them at risk of developing infections that may be life-threatening. Stem cell transplant can cure CGD but transplanting stem cells donated by other people can have serious complications. In addition, not everyone has a matched donor. Another approach is a type of gene therapy that involves base-editing to correct the mutation in a person s own stem cells. Researchers want to know if the base-edited stem cells can improve the white cells' functioning and result in fewer CGD-related infections. Objective: To learn if base-edited stem cells will improve white blood cells' ability to fight against infections in people with CGD. Eligibility: Males aged 18 years and older with X-linked CGD. Design: This is a non-randomized study. Participants with the specific mutation under study will be screened during the initial phase. During the development phase, participants will undergo apheresis to collect stem cells for base-editing correction of the mutation. During the treatment phase, participants will receive the base-edited cells after chemotherapy with busulfan. Participants will remain in the hospital until their immunity recovers. Follow-up visits will continue for 15 years.
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Development Status
Active
Indication
X-Linked Chronic Granulomatous Disease
Disease Ontology Term
DOID:0070195
Compound Name
Base-edited autologous CD34+ cells
Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Funder Type
NIH
Recruitment Status
Suspended
Enrollment Count
1
Results Posted
Not Available
Therapy Information
Target Gene/Variant
CYBB c.676 C>T
Therapy Type
Gene editing
Therapy Route
Ex-vivo
Mechanism of Action
Mutation correction
Route of Administration
Intravenous
Drug Product Type
Autologous cells
Target Tissue/Cell
CD34+ cells
Delivery System
Electroporation
Vector Type
none
Editor Type
ABE8e-SpRY
Dose 1
Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2024-03-21
Completion Date
2032-12-31
Last Update
2025-04-20
Participation Criteria
Eligible Age
18 Years - 75 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
1
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Trial stoppage rules triggered due to a serious adverse event
Resources/Links
Preclinical Publications
High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease