Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease
Condition(s) studied
Investigational drug(s) / intervention(s)
EN-374: Single dose of EN-374 administered by intravenous infusion after mobilization and followed by enrichment
Study 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
Eligibility
Primary outcome measure(s)
- Safety of EN-374 — From start of mobilization until Month 12
Incidence rate across all age groups of: * treatment-emergent adverse events (TEAEs) * treatment-related TEAEs (TRAEs) * serious adverse events (SAEs)
Trial sites (9)
| Facility | City | Region | Status |
|---|---|---|---|
| University of California, Los Angeles | Los Angeles | California | |
| University of California, San Francisco | San Francisco | California | |
| Johns Hopkins All Children's Hospital | St. Petersburg | Florida | |
| Boston Children's Hospital | Boston | Massachusetts | |
| University of Minnesota | Minneapolis | Minnesota | |
| Columbia University Irving Medical Center, Morgan Stanley Children's Hospital | New York | New York | |
| Duke University | Durham | North Carolina | |
| University of Utah, Primary Children's Hospital | Salt Lake City | Utah | |
| University College London Hospital | London | United Kingdom |
More Ensoma trials in the UK
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06876363 on ClinicalTrials.gov ↗ ← All trials in the UK