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NCT06876363 | NOT YET RECRUITING | X-Linked Chronic Granulomatous Disease

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

Condition or disease

X-Linked Chronic Granulomatous Disease

Intervention/treatment

EN-374

Phase

PHASE1

PHASE2

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 caused by mutations in the CYBB gene. 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.

Study Type : INTERVENTIONAL
Estimated Enrollment : 15 participants
Masking : NONE
Primary Purpose : TREATMENT
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
Actual Study Start Date : 2025-07
Estimated Primary Completion Date : 2027-12
Estimated Study Completion Date : 2027-12

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 3 Months
Sexes Eligible for Study: MALE
Accepts Healthy Volunteers:
Criteria
Inclusion 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
  • * An anti-EN-374 capsid antibody titer below an established threshold (ie, negative)
  • * Adequate organ function
Exclusion Criteria
  • * 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
Study of EN-374 Gene Therapy in Participants with X-Linked Chronic Granulomatous Disease

Location Details

NCT06876363

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