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Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) (OPTIRPE65)

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02781480
Recruitment Status : Completed
First Posted : May 24, 2016
Results First Posted : April 8, 2021
Last Update Posted : May 6, 2021
Sponsor:
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Study Description
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Brief Summary:
A clinical trial of AAV2/5 vector for patients with Defects in RPE65

Condition or disease Intervention/treatment Phase
Leber Congenital Amaurosis Biological: AAV RPE65 Phase 1 Phase 2

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 Design
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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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Low dose AAV-RPE65
Subretinal administration of a single low dose of range AAV-RPE65
 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


Outcome Measures
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Primary Outcome Measures :
  1. 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).


Eligibility Criteria
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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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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): NCT02781480


Locations
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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
Sponsors and Collaborators
MeiraGTx UK II Ltd
  Study Documents (Full-Text)

Documents provided by MeiraGTx UK II Ltd:
Study Protocol and Statistical Analysis Plan  [PDF] June 11, 2018

More Information
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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: May 6, 2021
Last Verified: April 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Blindness
Leber Congenital Amaurosis
Vision Disorders
Sensation Disorders
Neurologic Manifestations
 Nervous System Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Diseases