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4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
NCT04517149 · View on ClinicalTrials.gov ↗
Study 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 retinitis pigmentosa (XLRP) due to mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR).
Conditions Studied
Interventions
- OTHER Observational
- BIOLOGICAL 4D-125 IVT Injection
Study Locations (8)
Colorado
- University of Colorado — Aurora
Florida
- Vitreo Retinal Associates — Gainesville
Michigan
- University of Michigan Kellogg Eye Center — Ann Arbor
New York
- Columbia University Medical Center/Edward Harkness Eye Institute — New York
North Carolina
- Duke University Eye Center/Dept. of Ophthalmology — Durham
Oregon
- Casey Eye Institute, Oregon Health and Science University — Portland
Texas
- Retina Foundation of the Southwest — Dallas
Utah
- University of Utah John A. Moran Eye Center — Salt Lake City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 21 participants |
| Start Date | 2020-06-09 |
| Est. Completion | 2029-05 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04517149
The ClinicalTrials.gov registry entry for NCT04517149 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 21 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is 4D Molecular Therapeutics, which has 36 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with X-Linked Retinitis Pigmentosa appearing as the primary indexed condition, and to 2 interventions — of which Observational is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04517149 reports 8 study locations spanning 8 distinct geographic areas — top geographies include Colorado, Florida, Michigan. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04517149 about?
NCT04517149 is a clinical study titled "4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)". 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 retinitis pigmentosa (XLRP) due to mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPG...
What is the current status of trial NCT04517149?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 21 participants. The study started on 2020-06-09. Estimated completion is 2029-05.
What conditions does trial NCT04517149 study?
This clinical trial studies the following conditions: X-Linked Retinitis Pigmentosa. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04517149?
The interventions under investigation include: Observational (OTHER), 4D-125 IVT Injection (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04517149?
This trial is sponsored by 4D Molecular Therapeutics, which has 36 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04517149 being conducted?
This trial has 8 study locations across Colorado, Florida, Michigan, New York, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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