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Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)

NCT02946879 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 15

Last updated 2024-04-11

No results posted yet for this study

Summary

This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65.

Conditions

  • Leber Congenital Amaurosis (LCA)
  • Eye Diseases
  • Eye Diseases, Hereditary
  • Retinal Diseases

Interventions

BIOLOGICAL

AAV OPTIRPE65

comparison of different doses of AAV RPE65

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
2016-11-30
Primary Completion
2023-07-31
Completion
2023-07-31

Countries

  • United States
  • United Kingdom

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 NCT02946879 on ClinicalTrials.gov