Status:
ACTIVE_NOT_RECRUITING
4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
Lead Sponsor:
4D Molecular Therapeutics
Conditions:
X-Linked Retinitis Pigmentosa
Eligibility:
MALE
12+ years
Phase:
PHASE1
PHASE2
Brief Summary
This is a Phase 1/2 multicenter study with two parallel parts: an observational natural history cohort and an open-label, prospective interventional trial in males with non-syndromic X-linked retiniti...
Detailed Description
This Phase 1/2 study will gather data in an observational phase Natural History Cohort to further characterize and evaluate natural disease progression in male patients with genetically-confirmed X-li...
Eligibility Criteria
Inclusion
- Natural History Key Inclusion Criteria:
- Male, ≥ 6 years of age at the time of informed consent
- Hemizygous non-syndromic RPGR mutation confirmed by genetic testing
- Interventional Key Inclusion Criteria:
- Male, ≥12 years of age
- Hemizygous non-syndromic RPGR mutation confirmed by genetic testing
- Phase 1 Dose Exploration: At least one eye amenable to IVT injection and BCVA ≤ 78 ETDRS letters (\~20/32) and ≥ 34 ETDRS letters (\~20/200)
- Phase 2 Dose Expansion: At least one eye amenable to IVT injection AND both eyes must have BCVA ≥ 34 ETDRS letters (\~20/200)
- Key Exclusion Criteria (all cohorts)
- Patient has previously received any AAV treatment
- Pre-existing eye conditions or surgical complications that would preclude participation in an interventional clinical trial or interfere with the interpretation of study endpoints
Exclusion
Key Trial Info
Start Date :
June 9 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2029
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT04517149
Start Date
June 9 2020
End Date
May 1 2029
Last Update
March 21 2025
Active Locations (8)
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1
University of Colorado
Aurora, Colorado, United States, 80045
2
Vitreo Retinal Associates
Gainesville, Florida, United States, 32607
3
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, United States, 48105
4
Columbia University Medical Center/Edward Harkness Eye Institute
New York, New York, United States, 10032