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

Launched by ENSOMA · Mar 10, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new gene therapy called EN-374 for men with X-Linked Chronic Granulomatous Disease (X-CGD), a condition that affects the immune system and makes it hard for the body to fight infections. The main goals of the trial are to see if EN-374 is safe and how well it helps the body produce healthy immune cells called neutrophils, which are important for fighting off infections.

To participate in this trial, men must be at least 18 years old during the initial part and at least 3 months old for later stages, have a diagnosis of X-CGD, and have experienced serious infections or chronic inflammation. They should not have a suitable donor for a bone marrow transplant and must agree to follow guidelines regarding contraception if they are sexually active. Participants will need to give informed consent and meet other health criteria. The trial is not yet open for enrollment, but it offers a potential new treatment option for those affected by this challenging condition.

Gender

MALE

Eligibility 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
• • 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