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

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ClinicalTrials.gov Identifier: NCT02946879
Recruitment Status : Recruiting
First Posted : October 27, 2016
Last Update Posted : July 16, 2019
Sponsor:
Collaborator:
Syne Qua Non Limited
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Study Description
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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
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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

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Groups and Cohorts
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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
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Primary Outcome Measures :
  1. 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 :
  1. Improvement in the retinal function [ Time Frame: 5 years ]
    Improvements in visual function as assessed by visual assessment

  2. Improvement in the visual function [ Time Frame: 5 years ]
    Improvements in retinal function as assessed by visual assessment

  3. 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
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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
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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
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Contact: MeiraGTx UK II Ltd +44 (0)20 3866 4320 ocularinfo@meiragtx.com

Locations
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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
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Responsible Party: MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier: NCT02946879    
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:
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Blindness
Eye Diseases
Retinal Diseases
Retinal Dystrophies
Leber Congenital Amaurosis
Eye Diseases, Hereditary
 Genetic Diseases, Inborn
Retinal Degeneration
Vision Disorders
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases