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4D-125 for Retinitis Pigmentosa

Phase 1 & 2
Waitlist Available
Research Sponsored by 4D Molecular Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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)
Male, ≥ 6 years of age at the time of informed consent
Screening 3 weeks
Treatment Varies
Follow Up 24 months to 60 months
Awards & highlights

Study Summary

This trial is studying a treatment for non-syndromic X-linked retinitis pigmentosa, a disease that causes blindness.

Who is the study for?
This trial is for males aged 12 and older with X-linked retinitis pigmentosa (XLRP) due to RPGR gene mutations. Participants must have at least one eye suitable for injection and a certain level of vision measured by ETDRS letters. Those under phase 1 need vision between ~20/32 and ~20/200, while phase 2 requires both eyes to see at least ~20/200.Check my eligibility
What is being tested?
The study is testing the safety and effects of a new treatment called '4D-125 IVT Injection' in two parts: observing the natural progression of XLRP in some participants, while others receive the experimental injection directly into their eyes to assess its impact on their condition.See study design
What are the potential side effects?
As this is an early-phase trial for an investigational drug, specific side effects are not listed but may include typical risks associated with intravitreal injections such as eye inflammation, infection risk increase, possible visual disturbances or discomfort.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
I have at least one eye that can be treated with an injection and my vision is between 20/32 and 20/200.
I am a male and at least 6 years old.
My genetic test confirmed I have an RPGR mutation.


Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months to 60 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months to 60 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence and severity of TEAEs and serious adverse events (SAEs), including clinically significant changes in safety parameters

Trial Design

3Treatment groups
Experimental Treatment
Group I: ObservationalExperimental Treatment1 Intervention
Natural History
Group II: 4D-125 Dose ExplorationExperimental Treatment1 Intervention
Dose 1 and Dose 2 4D-125 will be administered at the assigned dose level as a single dose, IVT injection on Day 1. The contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent.
Group III: 4D-125 Dose ExpansionExperimental Treatment1 Intervention
4D-125 will be administered at the assigned dose level as a single dose, IVT injection on Day 1 in both adults and pediatric participants. For adult participants only, the contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent.

Find a Location

Who is running the clinical trial?

4D Molecular TherapeuticsLead Sponsor
7 Previous Clinical Trials
352 Total Patients Enrolled
Robert Kim, MDStudy Director4D Molecular Therapeutics
2 Previous Clinical Trials
70 Total Patients Enrolled
Chyong Nien, MDStudy Director4D Molecular Therapeutics
1 Previous Clinical Trials
150 Total Patients Enrolled

Media Library

4D-125 IVT Injection (Unknown) Clinical Trial Eligibility Overview. Trial Name: NCT04517149 — Phase 1 & 2
Retinitis Pigmentosa Research Study Groups: 4D-125 Dose Expansion, Observational, 4D-125 Dose Exploration
Retinitis Pigmentosa Clinical Trial 2023: 4D-125 IVT Injection Highlights & Side Effects. Trial Name: NCT04517149 — Phase 1 & 2
4D-125 IVT Injection (Unknown) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04517149 — Phase 1 & 2
Retinitis Pigmentosa Patient Testimony for trial: Trial Name: NCT04517149 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many participants have been accepted into this clinical research endeavor?

"Affirmative. Per the clinicaltrials.gov page, this medical trial is currently enlisting participants after being posted on June 9th 2020 and recently edited on August 18th 2022. 43 individuals need to be gathered from 8 different sites for the study's completion."

Answered by AI

Is this clinical experiment actively accepting participants?

"Clinicaltrials.gov indicates that this study is seeking enrollees and was initially made available on June 9th, 2020 with the last update occuring on August 18th 2022."

Answered by AI

How many sites offer access to this trial?

"Participants may select from 8 research centres, such as the Casey Eye Institute and Oregon Health and Science University in Portland. Additionally, there are Retina Foundation of the Southwest located in Dallas and Duke University Eye Center/Dept. of Ophthalmology in Durham among several other options available."

Answered by AI

Who else is applying?

What state do they live in?
Puerto Rico
How old are they?
18 - 65
What site did they apply to?
University of Colorado
University of Utah John A. Moran Eye Center
Retina Foundation of the Southwest
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?

Why did patients apply to this trial?

I was recommended by Vista Trial. I have not had any treatment for retinitis and I do not want to loose my eyesight completely.
PatientReceived 2+ prior treatments

What questions have other patients asked about this trial?

What other information do you need from me to start in the trial?
PatientReceived no prior treatments

How responsive is this trial?

Typically responds via
Phone Call
Most responsive sites:
  1. Columbia University Medical Center/Edward Harkness Eye Institute: < 48 hours
Average response time
  • < 2 Days
~7 spots leftby Jun 2026