NCT02946879

Brief 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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Nov 2016

Longer than P75 for all trials

Geographic Reach
2 countries

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 22, 2016

Completed
3 months until next milestone

First Posted

Study publicly available on registry

October 27, 2016

Completed
5 days until next milestone

Study Start

First participant enrolled

November 1, 2016

Completed
6.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2023

Completed
Last Updated

April 11, 2024

Status Verified

April 1, 2024

Enrollment Period

6.7 years

First QC Date

July 22, 2016

Last Update Submit

April 10, 2024

Conditions

Leber Congenital Amaurosis (LCA)Eye DiseasesEye Diseases, HereditaryRetinal Diseases

Outcome Measures

Primary Outcomes (1)

  • Incidence of Adverse Events related to the treatment

    Safety is defined as the absence of ATIMP-related safety events

    5 years

Secondary Outcomes (3)

  • Improvement in the retinal function

    5 years

  • Improvement in the visual function

    5 years

  • Improvement in quality of life

    5 years

Study Arms (3)

Low dose AAV OPTIRPE65

subretinal administration of a single low dose of AAV RPE65

Biological: AAV OPTIRPE65

Intermediate dose AAV OPTIRPE65

subretinal administration of a single intermediate dose of AAV RPE65

Biological: AAV OPTIRPE65

High dose AAV OPTIRPE65

subretinal administration of a single highdose of AAV RPE65

Biological: AAV OPTIRPE65

Interventions

AAV OPTIRPE65BIOLOGICAL

comparison of different doses of AAV RPE65

High dose AAV OPTIRPE65Intermediate dose AAV OPTIRPE65Low dose AAV OPTIRPE65

Eligibility Criteria

Age3 Years - 100 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

The patient population are those with Leber Congenital Amaurosis (LCA) with gene mutation RPE65 who have participated in the OPTIRPE65 trial.

You may qualify if:

  • Were enrolled and treated in the prior open-label, Phase I/II, dose escalation study involving intraocular administration of AAV2/5-OPTIRPE65

You may not qualify if:

  • Individuals will be excluded if they are unwilling or unable to meet with the requirements of the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Kellogg Eye Center, University of Michigan Health

Ann Arbor, Michigan, 48105, United States

Location

Moorfields Eye Hospital NHS Foundation Trust

London, United Kingdom

Location

MeSH Terms

Conditions

Leber Congenital AmaurosisEye DiseasesEye Diseases, HereditaryRetinal Diseases

Condition Hierarchy (Ancestors)

Genetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 22, 2016

First Posted

October 27, 2016

Study Start

November 1, 2016

Primary Completion

July 1, 2023

Study Completion

July 1, 2023

Last Updated

April 11, 2024

Record last verified: 2024-04

Locations

GB(1)US(1)