Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) (OPTIRPE65)
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ClinicalTrials.gov Identifier: NCT02781480 |
Recruitment Status :
Completed
First Posted : May 24, 2016
Results First Posted : April 8, 2021
Last Update Posted : July 12, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Leber Congenital Amaurosis | Biological: AAV RPE65 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 15 participants |
Allocation: | Non-Randomized |
Intervention Model: | Sequential Assignment |
Intervention Model Description: | AAV2/5-OPTIRPE65 |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA) |
Study Start Date : | April 2016 |
Actual Primary Completion Date : | December 2018 |
Actual Study Completion Date : | December 2018 |

Arm | Intervention/treatment |
---|---|
Experimental: Low dose AAV-RPE65
Subretinal administration of a single low dose of range AAV-RPE65
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Biological: AAV RPE65
Comparison of different dosages of AAV RPE65 |
Experimental: Intermediate dose AAV-RPE65
Subretinal administration of a single intermediate dose of range AAV-RPE65
|
Biological: AAV RPE65
Comparison of different dosages of AAV RPE65 |
Experimental: High dose AAV-RPE65
Subretinal administration of a single high dose of range AAV-RPE65
|
Biological: AAV RPE65
Comparison of different dosages of AAV RPE65 |
- Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event [ Time Frame: 6 months ]
Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related:
- Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more.
- Severe unresponsive inflammation.
- Infective endophthalmitis.
- Ocular malignancy.
- Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).

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 and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Key Inclusion Criteria:
- Aged 3 years or older
- Early-onset severe retinal dystrophy consistent with RPE65 deficiency
Key Exclusion Criteria:
- Females who are pregnant or breastfeeding
- Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.

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): NCT02781480
United States, Michigan | |
Kellogg Eye Centre, University of Michigan | |
Ann Arbor, Michigan, United States, 48105 | |
United Kingdom | |
Moorfields Eye Hospital NHS Foundation Trust | |
London, United Kingdom, EC1V 2PD |
Documents provided by MeiraGTx UK II Ltd:
Responsible Party: | MeiraGTx UK II Ltd |
ClinicalTrials.gov Identifier: | NCT02781480 |
Other Study ID Numbers: |
MGT003 |
First Posted: | May 24, 2016 Key Record Dates |
Results First Posted: | April 8, 2021 |
Last Update Posted: | July 12, 2021 |
Last Verified: | June 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Blindness Leber Congenital Amaurosis Vision Disorders Sensation Disorders Neurologic Manifestations |
Nervous System Diseases Eye Diseases Eye Diseases, Hereditary Retinal Diseases |