Status:
RECRUITING
Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease
Lead Sponsor:
Ensoma
Conditions:
X-Linked Chronic Granulomatous Disease
Eligibility:
MALE
3+ years
Phase:
PHASE1
PHASE2
Brief Summary
The goal of this clinical trial is to evaluate the safety and potential efficacy of the EN-374 treatment regimen and identify a dose level for further evaluation in participants with x-linked chronic ...
Detailed Description
Chronic granulomatous disease (CGD) is a rare primary immune deficiency disorder characterized by recurrent bacterial or fungal infections starting in infancy. The x-linked form of CGD (X-CGD) is caus...
Eligibility Criteria
Inclusion
- Male
- ≥ 18 years of age during dose escalation, then ≥ 3 months of age during dose expansion
- Diagnosis of X-CGD with DHR+ cells ≤ 5% and a pathogenic mutation in the CYBB gene
- History of at least 1 severe infection requiring medical intervention or chronic inflammatory disorder
- Does not have a suitable, available, and willing human leukocyte antigens (HLA)-matched (10/10) related donor
- Non-sterile male participants who are or may become sexually active with female partners of childbearing potential are required to use highly effective contraception
- Informed consent, with informed assent from capable participants
- Adequate organ function
Exclusion
- Active bacteremia or fungemia
- History of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C
- History or clinical evidence of any medical or social issues likely to put the participant at additional risk or to interfere with study conduct
- History of HSCT or granulocyte transfusions
- Known hypersensitivity to elements in the treatment regimen
- Undergone investigational gene therapy
- Treated with another investigational drug product within 30 days before screening
- Unable to comply with the visits and requirements of the protocol as determined by the Investigator
Key Trial Info
Start Date :
August 5 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2027
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT06876363
Start Date
August 5 2025
End Date
December 1 2027
Last Update
November 5 2025
Active Locations (4)
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1
University of Minnesota
Minneapolis, Minnesota, United States, 55454
2
Columbia University Irving Medical Center, Morgan Stanley Children's Hospital
New York, New York, United States, 10032
3
Duke University
Durham, North Carolina, United States, 27710
4
University of Utah, Primary Children's Hospital
Salt Lake City, Utah, United States, 84113