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

This study is currently recruiting participants.
Verified September 2017 by MeiraGTx UK II Ltd
Sponsor:
ClinicalTrials.gov Identifier:
NCT02946879
First Posted: October 27, 2016
Last Update Posted: September 11, 2017
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.
Collaborator:
Syne Qua Non Limited
Information provided by (Responsible Party):
MeiraGTx UK II Ltd
  Purpose
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 Intervention
Leber Congenital Amaurosis (LCA) Eye Diseases Eye Diseases, Hereditary Retinal Diseases Biological: AAV OPTIRPE65

Study Type: Observational
Study Design: 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)

Resource links provided by NLM:


Further study details as provided by MeiraGTx UK II Ltd:

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


Estimated Enrollment: 27
Study Start Date: November 2016
Estimated Study Completion Date: April 2023
Estimated Primary Completion Date: April 2023 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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

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.
  Eligibility

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.


Ages Eligible for Study:   3 Years to 100 Years   (Child, Adult, Senior)
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
Contact: Julie Bakobaki, MSc +44 (0)20 3866 4320 julie.bakobaki@meiragtx.com
Contact: Anna Morka, MSc 44 (0)20 3725 2350 anna.morka@meiragtx.com

Locations
United Kingdom
Moorfields Eye Hospital NHS Foundation Trust Recruiting
London, United Kingdom
Contact: Sophie Connor    02075662821    Sophie.Connor@moorfields.nhs.uk   
Contact: Neruban Kumaran, Dr       neruban.kumaran@moorfields.nhs.uk   
Principal Investigator: James Bainbridge, Prof         
Sponsors and Collaborators
MeiraGTx UK II Ltd
Syne Qua Non Limited
Investigators
Principal Investigator: James Bainbridge, Professor University College, London
  More 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 Submitted: July 22, 2016
First Posted: October 27, 2016
Last Update Posted: September 11, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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