Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
NCTID
NCT06851767
(View at clinicaltrials.gov)
Description
Background: X-linked severe combined immunodeficiency (XSCID) is a rare inherited disorder that affects the immune system. It is caused by a change in the IL2RG gene. Researchers are investigating a new type of gene therapy for people with XSCID. This technique, called base-edited stem cell transplants, involves collecting a person s own stem cells, editing the genes to repair IL2RG gene, and returning the edited cells to the person. Objective: To test base-edited stem cell transplants in people with XSCID. Eligibility: People aged 3 years and older with XSCID. Design: Participants will be screened. They will have a physical exam. They may give blood, urine, and stool samples. They may have tests of their heart and lung function. They may have fluid and cells drawn from their bone marrow. Participants will undergo apheresis. Blood will be taken from the body through a needle inserted into 1 arm. The blood will pass through a machine that separates out the stem cells. The remaining blood will be returned to the body through a different needle. The collected stem cells will undergo gene editing. Participants will be admitted to the hospital 1 week before treatment. They will receive a central line: A flexible tube will be inserted into a large vein. This tube will be used to administer drugs and draw blood during their stay. They will receive drugs to prepare their bodies for the treatment. The base-edited stem cells will be infused through the central line. Participants will remain in the hospital for at least 3 weeks while they recover. Follow-up visits will continue for 15 years.
(Show More)
Development Status
Active
Indication
X-linked Severe Combined Immunodeficiency (XSCID)
Disease Ontology Term
DOID:0060013
Compound Name
BE-HSPC-IL2RG
Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Funder Type
NIH
Recruitment Status
Not yet recruiting
Enrollment Count
18
Results Posted
Not Available
Therapy Information
Target Gene/Variant
IL2RG (p.Q144X)
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
Undisclosed
Vector Type
Editor Type
base editor
Dose 1
>5E6 cells/kg
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2025-02-26
Completion Date
2034-12-31
Last Update
2025-04-23
Participation Criteria
Eligible Age
3 Years - 99 Years
Standard Ages
Child, 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
Resources/Links
Resources/Links
No External Links Available.