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Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene

NCT06291935 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2025-03-28

No results posted yet for this study

Summary

The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa. The main questions the study aims to answer are:

* What is the best tolerated dose and are there any side effects, in particular any inflammatory reactions post drug administration?
* Are there any early signs of efficacy on visual function?

Participants will be administered a single intravitreal dose of VG901 into the most affected eye through a syringe and followed up for a year to monitor safety and efficacy. There will be two cohorts of participants in this study. Study Cohort 1 will receive the low dose and Study Cohort 2 will receive the high dose as specified in the Protocol.

Conditions

Interventions

DRUG

VG901

Administered as specified in the treatment arm. Study Cohort 1 - Low dose; Study Cohort 2 - High dose Other Names: Gene Therapy (AAV2.NN-CNGA1)

Sponsors & Collaborators

  • VeonGen Therapeutics GmbH

    lead INDUSTRY

Principal Investigators

  • Katarina Stingl · Center for Ophthalmology, University of Tuebingen

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-09-01
Primary Completion
2026-04-30
Completion
2026-04-30

Countries

  • Germany

Study Locations

More Related Trials

Entities

Diseases

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 NCT06291935 on ClinicalTrials.gov