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Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

NCT03316560 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 29

Last updated 2024-05-20

No results posted yet for this study

Summary

This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.

Conditions

  • X-Linked Retinitis Pigmentosa

Interventions

BIOLOGICAL

rAAV2tYF-GRK1-RPGR

Adeno-associated virus vector expressing a human RPGR gene

Sponsors & Collaborators

  • Beacon Therapeutics

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
6 Years
Max Age
50 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-04-16
Primary Completion
2023-11-09
Completion
2025-03-31
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 NCT03316560 on ClinicalTrials.gov