Study Details
Testing the Drug EN-374 for People with X-Linked Chronic Granulomatous Disease
(IRB#: IRB_00189912)
X-linked chronic Granulomatous Disease is a rare genetic disorder that impacts the ability of white blood cells to fight off certain infections. This study will test a drug called EN-374 to treat people with this disease. The study wants to see if the drug will help people with the disease and is safe. People in the study will receive the drug over a period of 14 months. This includes frequent in-person visits to the study clinic and up to 4 hospitalizations to receive medications. People will be paid for participation.
- Male Only
- All Ages
- Volunteers with special conditions
- In Person
- Paid
Who can participate?
Gender: Male Only
Age: All Ages
Volunteers: Volunteers with special conditions
Location: In Person
Inclusion Criteria
- Ages 3 months to 18 years and older
- History of at least 1 severe infection requiring medical intervention or chronic inflammatory disorder
- Diagnosis of X-CGD with DHR+ cells with specific criteria and a pathogenic mutation in the CYBB gene
- Adequate organ function
- Must use specific birth control if childbearing age
Exclusion Criteria
- History of any medical or social issues that increase risk or interfere with full study participation
- History of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C
- History of HSCT or granulocyte transfusions
- Known hypersensitivity to elements in the treatment
- Undergone investigational gene therapy or treated with another investigational drug product within 30 days before participation
Will I be paid for my time?
Yes
IRB#: IRB_00189912
PI: AHMAD RAYES
Department: PEDIATRIC HEMATOLOGY/ONCOLOGY
Approval Date: 2025-08-21 06:00:00
Specialties: Pediatric Hematology & Oncology, Pediatric Immunology
I am Interested