MedTrace Logo

Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3

NCT03278873 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 34

Last updated 2025-06-11

Study results available
· View outcomes & findings →

Summary

This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia associated with defects in CNGB3 who participated in a clinical trial in which they received AAV-CNGB3 retinal gene therapy.

Conditions

  • Achromatopsia

Interventions

BIOLOGICAL

Prior exposure to AAV-CNGA3 or AAV-CNGB3

Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively.

Sponsors & Collaborators

  • MeiraGTx UK II Ltd

    lead INDUSTRY

Eligibility

Min Age
3 Years
Max Age
100 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-06-29
Primary Completion
2024-04-04
Completion
2024-04-04
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

More Related Trials

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 NCT03278873 on ClinicalTrials.gov