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Natural History of Patients With Inherited Retinal Diseases Due to Mutations in RPE65 Gene

NCT04525261 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 60

Last updated 2023-11-29

No results posted yet for this study

Summary

Rationale:

In preparation for treatment with gene therapy, this study is being conducted in order to investigate the natural history of Inherited Retinal Dystrophies (IRDs) due to mutations in RPE65 gene. Such a study will help identify suitable patients for therapeutic intervention.

Methodology:

This is a multicenter retrospective, descriptive chart review study designed to assess retinal structure and function in subjects with IRDs due to mutation in RPE65 gene by visual acuity, visual field measurements, Optical Coherence Tomography (OCT), and a number of other vision-related assessments.

Conditions

  • Leber Congenital Amaurosis 2
  • Retinitis Pigmentosa 20

Sponsors & Collaborators

  • Retina Italia Onlus

    collaborator UNKNOWN

  • University of Campania Luigi Vanvitelli

    lead OTHER

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-05-01
Primary Completion
2020-07-31
Completion
2020-07-31

Countries

  • Italy

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 NCT04525261 on ClinicalTrials.gov