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Gene Therapy for Achromatopsia (CNGA3)

NCT03758404 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2022-12-01

Study results available
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Summary

A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia

Conditions

  • Achromatopsia

Interventions

BIOLOGICAL

adeno-associated virus vector AAV- CNGA3

Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene

Sponsors & Collaborators

  • Janssen Research & Development, LLC

    collaborator INDUSTRY

  • MeiraGTx UK II Ltd

    lead INDUSTRY

Principal Investigators

  • James Bainbridge · Chief Investigator

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-08-12
Primary Completion
2021-06-10
Completion
2021-06-10
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

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 NCT03758404 on ClinicalTrials.gov