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Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
Sponsor: 4D Molecular Therapeutics
Summary
This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM).
Official title: Phase 1 Open-Label, Dose-Escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia
Key Details
Gender
MALE
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
13
Start Date
2020-06-02
Completion Date
2027-08-31
Last Updated
2025-05-22
Healthy Volunteers
No
Conditions
Interventions
4D-110
4D-110 drug product developed for gene therapy which comprises an AAV capsid variant (4D-R100) carrying a transgene encoding a codon-optimized human CHM gene.
Locations (2)
Retina Foundation of the Southwest
Dallas, Texas, United States
Moran Eye Center, University of Utah
Salt Lake City, Utah, United States