🏛️ Verified NIH Record • Updated 2025-03-19
4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) (NCT04517149)
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-125 in Males With X-linked Retinitis Pigmentosa (XLRP) Caused by Mutations in the RPGR Gene
🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic Sponsor: 4D Molecular Therapeutics
🩺 Plain-English Summary (8th-Grade Level)
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).
Who can join: Review the eligibility criteria below.
Location: University of Colorado — Aurora, Colorado
Age range: 12 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (8 Sites)
- University of Colorado — Aurora, Colorado
- Vitreo Retinal Associates — Gainesville, Florida
- University of Michigan Kellogg Eye Center — Ann Arbor, Michigan
- Columbia University Medical Center/Edward Harkness Eye Institute — New York, New York
- Duke University Eye Center/Dept. of Ophthalmology — Durham, North Carolina
- Casey Eye Institute, Oregon Health and Science University — Portland, Oregon
- Retina Foundation of the Southwest — Dallas, Texas
- University of Utah John A. Moran Eye Center — Salt Lake City, Utah
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in X-Linked Retinitis Pigmentosa
- Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations — Durham, North Carolina
- Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene — La Jolla, California
- A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP — Scottsdale, Arizona
- A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP) — Boston, Massachusetts
- A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE) — Jacksonville, Florida
Explore by condition: All X-Linked Retinitis Pigmentosa clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.