This study looks at a gene therapy called EN-374 in people with X-linked chronic granulomatous disease (X-CGD). Males with a disease-causing mutation in the CYBB gene and a history of at least one severe infection or chronic inflammatory disorder can take part. It is a Phase 1/Phase 2 study and is currently recruiting participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Male * ≥ 18 years of age during dose escalation, then ≥ 3 months of age during dose expansion * Diagnosis of X-CGD with 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