Status:
ACTIVE_NOT_RECRUITING
Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
Lead Sponsor:
4D Molecular Therapeutics
Conditions:
Choroideremia
Eligibility:
MALE
18+ years
Phase:
PHASE1
Brief 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 wi...
Detailed Description
This is an open-label, Phase 1 study to evaluate safety and tolerability as well as preliminary efficacy of a single IVT injection of 4D-110 at three dose levels in male patients with genetically-conf...
Eligibility Criteria
Inclusion
- Key
- Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing
- Both eyes must have ≥ 34 ETDRS letters (\~20/200)
- Key
Exclusion
- Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye
- Patient has previously received any AAV treatment
- Ocular conditions or ocular media opacity in either eye that would preclude the planned treatment (i.e. IVT injection) or interfere with the interpretation of study endpoints (e.g. significant lens opacity)
Key Trial Info
Start Date :
June 2 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 31 2027
Estimated Enrollment :
13 Patients enrolled
Trial Details
Trial ID
NCT04483440
Start Date
June 2 2020
End Date
August 31 2027
Last Update
May 22 2025
Active Locations (2)
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1
Retina Foundation of the Southwest
Dallas, Texas, United States, 75251
2
Moran Eye Center, University of Utah
Salt Lake City, Utah, United States, 84132