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4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)

NCT04517149 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2025-03-21

No results posted yet for this 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

  • X-Linked Retinitis Pigmentosa

Interventions

BIOLOGICAL

4D-125 IVT Injection

4D-125 drug product developed for gene therapy, which comprises an AAV capsid variant (4D-R100) carrying a codon-optimized human Retinitis Pigmentosa GTPase Regulator transgene.

OTHER

Observational

Natural History

Sponsors & Collaborators

  • 4D Molecular Therapeutics

    lead INDUSTRY

Principal Investigators

  • Schonmei Lee, MD · 4D Molecular Therapeutics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
12 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-06-09
Primary Completion
2026-06-30
Completion
2029-05-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04517149 on ClinicalTrials.gov