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

This study is currently recruiting participants.
Verified September 2017 by MeiraGTx UK II Ltd
Sponsor:
ClinicalTrials.gov Identifier:
NCT02781480
First Posted: May 24, 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.
Information provided by (Responsible Party):
MeiraGTx UK II Ltd
  Purpose
A clinical trial of AAV2/5 vector for patients with Defects in RPE65

Condition Intervention Phase
Leber Congenital Amaurosis Biological: AAV RPE65 Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group 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)

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: 1 year ]
    Safety is defined as the absence of ATIMP-related safety events


Secondary Outcome Measures:
  • Improvement in visual function [ Time Frame: 6 months ]
    Improvements in visual function as assessed by visual assessment

  • Improvement in retinal function [ Time Frame: 6 months ]
    Improvements in retinal function as assessed by visual assessment

  • Improvement in Quality of Life [ Time Frame: 6 months ]
    Quality of life will be measured by QoL questionnaire


Estimated Enrollment: 27
Study Start Date: April 2016
Estimated Study Completion Date: October 2018
Estimated Primary Completion Date: October 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Senior)
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
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


Contacts
Contact: Julie Bakobaki julie.bakobaki@meiragtx.com
Contact: Praseeda Thaikalloor praseeda.thaikalloor@meiragtx.com

Locations
United Kingdom
Moorfields Eye Hospital NHS Foundation Trust Recruiting
London, United Kingdom, EC1V 2PD
Contact: James Bainbridge       j.bainbridge@ucl.ac.uk   
Contact: Neruban Kumaran       neruban.kumaran@moorfields.nhs.co.uk   
Principal Investigator: James Bainbridge         
Sub-Investigator: Neruban Kumaran         
Sponsors and Collaborators
MeiraGTx UK II Ltd
  More Information

Responsible Party: MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier: NCT02781480     History of Changes
Other Study ID Numbers: MGT003
First Submitted: April 28, 2016
First Posted: May 24, 2016
Last Update Posted: September 11, 2017
Last Verified: September 2017

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

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