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

Who is this study for? Males with non-syndromic X-linked retinitis pigmentosa due to mutations in retinitis pigmentosa GTPase regulator

What treatments are being studied? 4D-125

Status: Active_not_recruiting

Location: See all (8) locations...

Intervention Type: Biological, Other

Study Type: Interventional

Study Phase: Phase 1/Phase 2

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).

Eligibility

Participation Requirements

Sex: Male

Minimum Age: 12

Healthy Volunteers: f

View:

• Male, ≥ 6 years of age at the time of informed consent

• Hemizygous non-syndromic RPGR mutation confirmed by genetic testing

• Male, ≥12 years of age

• Hemizygous non-syndromic RPGR mutation confirmed by genetic testing

• 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)

• Phase 2 Dose Expansion: At least one eye amenable to IVT injection AND both eyes must have BCVA ≥ 34 ETDRS letters (\

⁃ 20/200)

Locations

United States

Colorado

University of Colorado

Aurora

Florida

Vitreo Retinal Associates

Gainesville

Michigan

University of Michigan Kellogg Eye Center

Ann Arbor

North Carolina

Duke University Eye Center/Dept. of Ophthalmology

Durham

New York

Columbia University Medical Center/Edward Harkness Eye Institute

New York

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

Time Frame

Start Date: 2020-06-09

Completion Date: 2029-05

Participants

Target number of participants: 21

Treatments

Experimental: 4D-125 Dose Exploration

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.

Experimental: 4D-125 Dose Expansion

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.

Other: Observational

Natural History

Authors

Paul Yang, Alessandro Iannaccone, Marc Mathias, Christine Kay, Paul Bernstein, Cagri Besirli

Related Therapeutic Areas

X-Linked Retinitis Pigmentosa (XLRP)

Retinitis Pigmentosa

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