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

Active Not RecruitingPhase 1, Phase 2

Sponsor: Ensoma

Conditions: X-Linked Chronic Granulomatous Disease

Interventions: EN-374

Countries: United States, United Kingdom

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

Eligibility overview

Sex: MALE

Age: 3 Months to —

Healthy volunteers: No

Study type: INTERVENTIONAL

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 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

Locations (9)

Los Angeles, California, United States
San Francisco, California, United States
St. Petersburg, Florida, United States
Boston, Massachusetts, United States
Minneapolis, Minnesota, United States
New York, New York, United States
Durham, North Carolina, United States
Salt Lake City, Utah, United States
London, United Kingdom