4D-125 IVT Injection for Retinitis Pigmentosa

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Retinitis Pigmentosa+2 More
4D-125 IVT Injection - Biological
Eligibility
Any Age
Male
What conditions do you have?
Select

Study Summary

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

Eligible Conditions
  • Retinitis Pigmentosa

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Retinitis Pigmentosa

Study Objectives

1 Primary · 0 Secondary · Reporting Duration: 24 months to 60 Months

Month 60
measure

Trial Safety

Safety Progress

1 of 3

Other trials for Retinitis Pigmentosa

Trial Design

2 Treatment Groups

4D-125 Dose Expansion
1 of 2
4D-125 Dose Exploration
1 of 2
Experimental Treatment

43 Total Participants · 2 Treatment Groups

Primary Treatment: 4D-125 IVT Injection · No Placebo Group · Phase 1 & 2

4D-125 Dose Expansion
Biological
Experimental Group · 1 Intervention: 4D-125 IVT Injection · Intervention Types: Biological
4D-125 Dose Exploration
Biological
Experimental Group · 1 Intervention: 4D-125 IVT Injection · Intervention Types: Biological

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 24 months to 60 months

Who is running the clinical trial?

4D Molecular TherapeuticsLead Sponsor
5 Previous Clinical Trials
176 Total Patients Enrolled
Robert Kim, MDStudy Director4D Molecular Therapeutics
2 Previous Clinical Trials
72 Total Patients Enrolled
Schonmei Lee, MDStudy Director4D Molecular Therapeutics
2 Previous Clinical Trials
122 Total Patients Enrolled
Chyong Nien, MDStudy Director4D Molecular Therapeutics
1 Previous Clinical Trials
65 Total Patients Enrolled

Eligibility Criteria

Age Any Age · Male Participants · 6 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
At least one eye with BCVA ≤ 78 ETDRS letters (~20/32) and ≥ 34 ETDRS letters (~20/200).
You have a hemizygous non-syndromic RPGR mutation.
You are male and you are 12 years of age or older.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 17th, 2021

Last Reviewed: October 9th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

Who else is applying?

What state do they live in?
Texas100.0%
How old are they?
18 - 65100.0%
What site did they apply to?
Retina Foundation of the Southwest100.0%
What portion of applicants met pre-screening criteria?
Met criteria100.0%