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 |
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Recruitment Status :
Recruiting
First Posted : October 27, 2016
Last Update Posted : December 18, 2017
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Sponsor:
MeiraGTx UK II Ltd
Collaborator:
Syne Qua Non Limited
Information provided by (Responsible Party):
MeiraGTx UK II Ltd
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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 |
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 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
| Group/Cohort | Intervention/treatment |
|---|---|
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Low dose AAV OPTIRPE65
subretinal administration of a single low dose of AAV RPE65
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Biological: AAV OPTIRPE65
comparison of different doses of AAV RPE65 |
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Intermediate dose AAV OPTIRPE65
subretinal administration of a single intermediate dose of AAV RPE65
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Biological: AAV OPTIRPE65
comparison of different doses of AAV RPE65 |
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High dose AAV OPTIRPE65
subretinal administration of a single highdose of AAV RPE65
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Biological: AAV OPTIRPE65
comparison of different doses of AAV RPE65 |
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
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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.
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: Praseeda Thaikalloor, MSc | praseeda.thaikalloor@meiragtx.com |
Locations
| 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
| 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: | December 18, 2017 |
| Last Verified: | December 2017 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Undecided |
Additional relevant MeSH terms:
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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 |