Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT04517149 · ClinicalTrials.gov registry record · Phase 1
4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
A Phase 1 study of X-Linked Retinitis Pigmentosa, sponsored by 4D Molecular Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 21
- Enrollment target
- 8
- Study locations
NCT04517149: Active Phase 1 study of X-Linked Retinitis Pigmentosa, sponsored by 4D Molecular Therapeutics.
NCT04517149 is a Phase 1 study of X-Linked Retinitis Pigmentosa that is active but no longer recruiting, run by 4D Molecular Therapeutics. The registered enrollment target is 21 participants, below the 56-participant average among 8 other X-Linked Retinitis Pigmentosa trials with a reported enrollment target (62% lower). The trial reports 8 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04517149, a Phase 1 study of X-Linked Retinitis Pigmentosa, is active but no longer recruiting, sponsored by 4D Molecular Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 21 participants
- Enrollment target
- 8
- Study locations
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 |
What the registry record for NCT04517149 still lists
NCT04517149 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 21 participants, a relatively small participant target, below the 56-participant average among 8 other X-Linked Retinitis Pigmentosa trials with a reported enrollment target (62% lower).
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.
NCT04517149 lists 8 locations in 8 states (Colorado, Florida, Michigan).
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.
What interventions are being tested in trial NCT04517149?
The interventions under investigation include: Observational (OTHER), 4D-125 IVT Injection (BIOLOGICAL).
Who is sponsoring clinical trial NCT04517149?
This trial is sponsored by 4D Molecular Therapeutics, which has 9 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
Similar trials for X-Linked Retinitis Pigmentosa
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04517149's enrollment target sits among peer trials
21 8th of 8 higher than 1 of 8 other X-Linked Retinitis Pigmentosa trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other X-Linked Retinitis Pigmentosa trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
ACTIVE NOT RECRUITING · Phase 1
-
A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112
COMPLETED · Phase 1
-
A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
ACTIVE NOT RECRUITING · Phase 2
-
A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
ACTIVE NOT RECRUITING · Phase 2
-
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
ACTIVE NOT RECRUITING · Phase 2
-
Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
ACTIVE NOT RECRUITING · Phase 3
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02447874 · 21 participants · Phase 1
Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease
- NCT03826992 · 21 participants · Phase 1
Venetoclax Combined With Vyxeos (CPX-351) for Participants With Relapsed or Refractory Acute Leukemia
- NCT04107077 · 21 participants · Phase 2
Phase II Study of the Effects of Laparoscopic Hyperthermic Intraperitoneal Chemotherapy (HIPEC) in Patients With Advanced Gastric Cancer
- NCT04158635 · 21 participants · Phase 1
Gemcitabine, Nab-Paclitaxel, and Bosentan for the Treatment of Unresectable Pancreatic Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI