RECRUITING

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

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

Conditions

X-Linked Chronic Granulomatous Disease
Talk to us

Related Conditions:

X-Linked Chronic Granulomatous Disease

Study Overview

On this page

Study Details

Study overview

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

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

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

Condition
X-Linked Chronic Granulomatous Disease
Intervention / Treatment

-

Contacts and Locations

Minneapolis

University of Minnesota, Minneapolis, Minnesota, United States, 55454

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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

Ages Eligible for Study

3 Months to

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Ensoma,

Study Record Dates

2027-12