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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.

ClinicalTrials.gov Identifier: NCT02946879

Recruitment Status : Recruiting

First Posted : October 27, 2016

Last Update Posted : July 16, 2019

See Contacts and Locations

Sponsor:

Collaborator:

Syne Qua Non Limited

Information provided by (Responsible Party):

MeiraGTx UK II Ltd

Study Description

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.

Condition or disease	Intervention/treatment
Leber Congenital Amaurosis (LCA) Eye Diseases Eye Diseases, Hereditary Retinal Diseases	Biological: AAV OPTIRPE65

Detailed Description:

The follow up study is designed to collect data on longer-term safety and efficacy of AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial.

Study Design

Layout table for study information

Study Type :	Observational
Estimated Enrollment :	27 participants
Observational Model:	Cohort
Time Perspective:	Prospective
Official Title:	Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children With Retinal Dystrophy Owing to Defects in RPE65 (LCA2)
Study Start Date :	November 2016
Estimated Primary Completion Date :	April 2023
Estimated Study Completion Date :	April 2023

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: Leber congenital amaurosis Lenz microphthalmia syndrome Oculofaciocardiodental syndrome

MedlinePlus related topics: Genes and Gene Therapy

Genetic and Rare Diseases Information Center resources: Leber Congenital Amaurosis

U.S. FDA Resources

Groups and Cohorts

Group/Cohort	Intervention/treatment
Low dose AAV OPTIRPE65 subretinal administration of a single low dose of AAV RPE65	Biological: AAV OPTIRPE65 comparison of different doses of AAV RPE65
Intermediate dose AAV OPTIRPE65 subretinal administration of a single intermediate dose of AAV RPE65	Biological: AAV OPTIRPE65 comparison of different doses of AAV RPE65
High dose AAV OPTIRPE65 subretinal administration of a single highdose of AAV RPE65	Biological: AAV OPTIRPE65 comparison of different doses of AAV RPE65

Outcome Measures

Primary Outcome Measures :

Incidence of Adverse Events related to the treatment [ Time Frame: 5 years ]
Safety is defined as the absence of ATIMP-related safety events

Secondary Outcome Measures :

Improvement in the retinal function [ Time Frame: 5 years ]
Improvements in visual function as assessed by visual assessment
Improvement in the visual function [ Time Frame: 5 years ]
Improvements in retinal function as assessed by visual assessment
Improvement in quality of life [ Time Frame: 5 years ]
Improvement in the participant's quality of life which is measurable by QoL questionnaire

Eligibility Criteria

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information

Ages Eligible for Study:	3 Years to 100 Years (Child, Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No
Sampling Method:	Non-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.

Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02946879

Contacts

Layout table for location contacts

Contact: MeiraGTx UK II Ltd	+44 (0)20 3866 4320	ocularinfo@meiragtx.com

Locations

Layout table for location information

United Kingdom
Moorfields Eye Hospital NHS Foundation Trust	Recruiting
London, United Kingdom
Contact: Michel Michaelides michel.michaelides@ucl.ac.uk
Principal Investigator: James Bainbridge, Prof
Sub-Investigator: Michel Michaelides, Prof

Sponsors and Collaborators

MeiraGTx UK II Ltd

Syne Qua Non Limited

More Information

Layout table for additonal information

Responsible Party:	MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier:	NCT02946879 History of Changes
Other Study ID Numbers:	MGT004 2016-000898-20 ( EudraCT Number )
First Posted:	October 27, 2016 Key Record Dates
Last Update Posted:	July 16, 2019
Last Verified:	July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	Undecided

Additional relevant MeSH terms:

Layout table for MeSH terms

Blindness Retinal Diseases Leber Congenital Amaurosis Retinal Dystrophies Eye Diseases, Hereditary Genetic Diseases, Inborn Eye Diseases	Vision Disorders Sensation Disorders Neurologic Manifestations Nervous System Diseases Signs and Symptoms Retinal Degeneration

To Top