- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02781480
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) (OPTIRPE65)
June 17, 2021 updated by: MeiraGTx UK II Ltd
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)
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65.
ATIMP will be administered to one eye only in a single sub-retinal procedure
Study Type
Interventional
Enrollment (Actual)
15
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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London, United Kingdom, EC1V 2PD
- Moorfields Eye Hospital NHS Foundation Trust
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Michigan
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Ann Arbor, Michigan, United States, 48105
- Kellogg Eye Centre, University of Michigan
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
3 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Low dose AAV-RPE65
Subretinal administration of a single low dose of range AAV-RPE65
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Comparison of different dosages of AAV RPE65
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Experimental: Intermediate dose AAV-RPE65
Subretinal administration of a single intermediate dose of range AAV-RPE65
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Comparison of different dosages of AAV RPE65
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Experimental: High dose AAV-RPE65
Subretinal administration of a single high dose of range AAV-RPE65
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Comparison of different dosages of AAV RPE65
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event
Time Frame: 6 months
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Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related:
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6 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
April 1, 2016
Primary Completion (Actual)
December 1, 2018
Study Completion (Actual)
December 1, 2018
Study Registration Dates
First Submitted
April 28, 2016
First Submitted That Met QC Criteria
May 23, 2016
First Posted (Estimate)
May 24, 2016
Study Record Updates
Last Update Posted (Actual)
July 12, 2021
Last Update Submitted That Met QC Criteria
June 17, 2021
Last Verified
June 1, 2021
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- MGT003
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
UNDECIDED
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Leber Congenital Amaurosis
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ProQR TherapeuticsActive, not recruitingEye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders CongenitalUnited States, Belgium, Brazil, Canada, France, Germany, Italy, Netherlands, United Kingdom
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ProQR TherapeuticsTerminatedEye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders CongenitalUnited States, Belgium
-
University of PennsylvaniaNational Eye Institute (NEI)Active, not recruitingRetinal Diseases | Amaurosis of LeberUnited States
-
Eyecure Therapeutics Inc.Beijing Tongren HospitalUnknownLeber Congenital Amaurosis, Retinitis PigmentosaChina
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QLT Inc.CompletedLCA (Leber Congenital Amaurosis) | RP (Retinitis Pigmentosa)United States, Canada, Germany, Netherlands, United Kingdom
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ProQR TherapeuticsRecruitingEye Diseases | Neurologic Manifestations | Retinal Degeneration | Retinal Dystrophies | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders CongenitalBelgium, Brazil, Canada, Germany, Italy, Netherlands, United Kingdom
-
QLT Inc.CompletedLCA (Leber Congenital Amaurosis) | RP (Retinitis Pigmentosa)Canada, United States, Germany, Netherlands, United Kingdom
-
University of Campania "Luigi Vanvitelli"Retina Italia OnlusCompletedLeber Congenital Amaurosis 2 | Retinitis Pigmentosa 20Italy
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QLT Inc.CompletedLeber Congenital Amaurosis (LCA) | Retinitis Pigmentosa (RP)United States, Canada, Denmark, Germany, Netherlands, Switzerland, United Kingdom
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ProQR TherapeuticsCompletedLeber's Congenital AmaurosisUnited States, Belgium
Clinical Trials on AAV RPE65
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MeiraGTx UK II LtdSyne Qua Non Limited; EMAS PharmaActive, not recruitingAchromatopsiaUnited Kingdom, United States
-
Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States
-
MeiraGTx UK II LtdSyne Qua Non Limited; EMAS PharmaCompleted
-
MeiraGTx UK II LtdSyne Qua Non LimitedCompletedEye Diseases | Retinal Diseases | Eye Diseases, Hereditary | Leber Congenital Amaurosis (LCA)United States, United Kingdom
-
Libella Gene TherapeuticsUnknown
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Libella Gene TherapeuticsUnknownEvaluation of Safety and Tolerability of Libella Gene Therapy for Critical Limb Ischemia: AAV- hTERTCritical Limb IschemiaColombia
-
National Eye Institute (NEI)Active, not recruiting
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Libella Gene TherapeuticsUnknownAlzheimer DiseaseColombia
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Lantu BiopharmaNot yet recruitingSpinal Muscular Atrophy
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University of OxfordUniversity College, London; Moorfields Eye Hospital NHS Foundation TrustCompleted