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

Part of paid clinical trials in Los Angeles, California.

Sponsor
Ensoma
Study ID
NCT06876363
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • X-Linked Chronic Granulomatous Disease

Eligibility Criteria

Sex
MALE
Age
3 Months - N/A
Healthy Volunteers
Not accepted

Interventions

  • EN-374 — GENETIC
    Single dose of EN-374 administered by intravenous infusion after mobilization and followed by enrichment

Study Details

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

Key Dates

Start date
Aug 5, 2025
Status verified
Apr 2026
Primary completion
Dec 31, 2027
Completion
Dec 31, 2027

Study Design

Enrollment
15 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: EN-374
    Single dose of EN-374 administered by intravenous infusion after mobilization and followed by enrichment

Primary Outcome Measure

Safety of EN-374 [ Time Frame: From start of mobilization until Month 12 ]

Central Contacts

Locations (8)

FacilityCityStateZIPSite coordinators
University of California, Los AngelesLos AngelesCalifornia90095
Bruck Habtemariam
[email protected]
310-794-0242
Augustine Fernandes, PhD
[email protected]
310-206-7802
Donald Kohn, MD (PRINCIPAL_INVESTIGATOR)
Gary Schiller, MD (SUB_INVESTIGATOR)
University of California, San FranciscoSan FranciscoCalifornia94158
Audrey Hernando, CCRP
[email protected]
415-502-2425
Morna Dorsey, MD, MS (PRINCIPAL_INVESTIGATOR)
Johns Hopkins All Children's HospitalSt. PetersburgFlorida33701
Anna Eidenberger, MS, ACRP-CP
[email protected]
727-767-2524
Laura LaBarre, MD
[email protected]
727-767-3989
Jennifer Leiding, MD (PRINCIPAL_INVESTIGATOR)
Boston Children's HospitalBostonMassachusetts02115
Emily Morris, MPH
[email protected]
617-632-1954
Susan Prockop, MD (PRINCIPAL_INVESTIGATOR)
University of MinnesotaMinneapolisMinnesota55454
Alli Travis, BSN, RN, BMTCN
[email protected]
612-625-6150
Christen Ebens, MD, MPH (PRINCIPAL_INVESTIGATOR)
Columbia University Irving Medical Center, Morgan Stanley Children's HospitalNew YorkNew York10032
Brianna Mayo, PNP, RN
[email protected]
212-305-2372
Joseph Oved, MD (PRINCIPAL_INVESTIGATOR)
Duke UniversityDurhamNorth Carolina27710
Linda Brown
[email protected]
919-613-1482
Talal Mousallem, MD (PRINCIPAL_INVESTIGATOR)
Kris Mahadeo, MD, MPH (PRINCIPAL_INVESTIGATOR)
University of Utah, Primary Children's HospitalSalt Lake CityUtah84113
Morgan Badgley, BS, CCRC
[email protected]
801-662-4812
Chelsie Tucker, BS, CRC
[email protected]
801-662-4813
Ahmad Rayes, MD, MSc (PRINCIPAL_INVESTIGATOR)

Find similar trials in Los Angeles, CA

By research site
University of California, Los Angeles· Los Angeles, CAUniversity of California, San Francisco· San Francisco, CAJohns Hopkins All Children's Hospital· St. Petersburg, FLBoston Children's Hospital· Boston, MAUniversity of Minnesota· Minneapolis, MNColumbia University Irving Medical Center, Morgan Stanley Children's Hospital· New York, NY

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