RECRUITING

Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease

Conditions

X-Linked Chronic Granulomatous Disease CGD X-CGD Chronic Granulomatous Disease Granulomatous Disease, Chronic Genetic Therapy Gene Therapy in vivo Gene Therapy Gene Addition Therapy in vivo Gene Addition Therapy Hematopoietic Stem Cell Gene Therapy in vivo Hematopoietic Stem Cell Gene Therapy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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 granulomatous disease. 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

Official Title

A Phase 1/2 Open-Label, Single-Ascending-Dose Study of EN-374, a Helper-Dependent Adenoviral-Based Gene Therapy, in Participants With X-Linked Chronic Granulomatous Disease

Quick Facts

Study Start:2025-08-05

Study Completion:2027-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06876363

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:3 Months

Sexes Eligible for Study:MALE

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* 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	* 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

Inclusion Criteria

Exclusion Criteria

* 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

* 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

Contacts and Locations

Study Contact

Andrew Dietz, MD, MSCR

CONTACT

617-766-3917

ddietz@ensoma.com

Study Locations (Sites)

University of Minnesota

Minneapolis, Minnesota, 55454

United States

Collaborators and Investigators

Sponsor: Ensoma

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-08-05

Study Completion Date2027-12

Study Record Updates

Study Start Date2025-08-05

Study Completion Date2027-12

Terms related to this study

Keywords Provided by Researchers

CGD
X-CGD
Chronic Granulomatous Disease
X-Linked Chronic Granulomatous Disease
Granulomatous Disease, Chronic
Genetic Therapy
Gene Therapy
in vivo Gene Therapy
Gene Addition Therapy
in vivo Gene Addition Therapy
Hematopoietic Stem Cell Gene Therapy
in vivo Hematopoietic Stem Cell Gene Therapy

Additional Relevant MeSH Terms

X-Linked Chronic Granulomatous Disease