Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease
NCT ID: NCT06876363
Last Updated: 2025-11-05
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
15 participants
INTERVENTIONAL
2025-08-05
2027-12-31
Brief Summary
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The main questions it aims to answer are:
* safety of the EN-374 treatment regimen
* effect of the EN-374 treatment regimen on the production of functional neutrophils with NADPH oxidase activity
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Detailed Description
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EN-374 is a helper-dependent adenoviral (HDAd)-based gene therapy in development for the treatment of X-CGD using an in vivo approach, which is administered by IV infusion, to genetically modify hematopoietic stem cells (HSCs) to express a wild-type CYBB gene. The EN-374 treatment regimen includes HSC mobilization, immune prophylaxis, EN-374 administration, and enrichment of genetically modified HSCs.
Adult participants with X-CGD will be enrolled into the dose-escalation part of the study. Following completion of the adult cohorts, then pediatric participants will be enrolled into the dose-expansion part of the study in decreasing age cohorts from ≥ 12 and \< 18 years of age, to ≥ 2 and \< 12 years of age, and finally to ≥ 3 months and \< 2 years of age.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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EN-374
Single dose of EN-374 administered by intravenous infusion after mobilization and followed by enrichment
EN-374
Single dose of EN-374 administered by intravenous infusion after mobilization and followed by enrichment
Interventions
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EN-374
Single dose of EN-374 administered by intravenous infusion after mobilization and followed by enrichment
Eligibility Criteria
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Inclusion Criteria
* ≥ 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 Criteria
* 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
3 Months
MALE
No
Sponsors
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Ensoma
INDUSTRY
Responsible Party
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Locations
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University of Minnesota
Minneapolis, Minnesota, United States
Columbia University Irving Medical Center, Morgan Stanley Children's Hospital
New York, New York, United States
Duke University
Durham, North Carolina, United States
University of Utah, Primary Children's Hospital
Salt Lake City, Utah, United States
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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EN-374-101
Identifier Type: -
Identifier Source: org_study_id
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