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Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) Retinitis Pigmentosa GTPase Regulator (RPGR)

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ClinicalTrials.gov Identifier: NCT03252847
Recruitment Status : Recruiting
First Posted : August 17, 2017
Last Update Posted : July 17, 2019
Sponsor:
Collaborators:
Syne Qua Non Limited
Bionical-Emas Pharma Ltd
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Study Description
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Brief Summary:
A clinical trial of AAV2 vector for patients with X-linked retinitis pigmentosa (XLRP)

Condition or disease Intervention/treatment Phase
X-Linked Retinitis Pigmentosa Genetic: AAV-RPGR Phase 1 Phase 2

Detailed Description:
This is an open-label phase I/II dose-escalation trial to determine the safety and efficacy of subretinal administration of AAV2 vector in participants with XLRP caused by mutations in RPGR.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description: Biological
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2-.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
Actual Study Start Date : July 14, 2017
Estimated Primary Completion Date : November 2020
Estimated Study Completion Date : November 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Low dose AAV-RPGR
Single, subretinal administration of low dose AAV-RPGR
 Genetic: AAV-RPGR
Open label, non-randomised, dose-escalation intervention followed by randomised dose confirmation against a control arm
Experimental: Intermediate dose AAV-RPGR
Single, subretinal administration of intermediate dose AAV-RPGR
 Genetic: AAV-RPGR
Open label, non-randomised, dose-escalation intervention followed by randomised dose confirmation against a control arm
Experimental: High dose AAV-RPGR
Single, subretinal administration of high dose AAV-RPGR
 Genetic: AAV-RPGR
Open label, non-randomised, dose-escalation intervention followed by randomised dose confirmation against a control arm


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of Adverse Events related to the sub retinal administration of AAV2-RPGR [ Time Frame: 18 months ]
    Safety is defined as the absence of ATIMP-related safety events


Secondary Outcome Measures :
  1. Improvement in visual function [ Time Frame: 18 months ]
    Improvements in visual function as assessed by ocular examination

  2. Improvement in retinal function [ Time Frame: 18 months ]
    Improvements in retinal function as assessed by retinal assessement

  3. Improvement in Quality of Life [ Time Frame: 18 months ]
    •Quality of life will be measured by QoL questionnaire


Eligibility Criteria
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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Key inclusion Criteria:

  • Are aged 5 years or older male
  • Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI)

Key exclusion Criteria:

• Have participated in another research study involving an investigational medicinal 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): NCT03252847


Contacts
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Contact: MeiraGTx UK II Ltd +44 (0)20 3866 4320 ocularinfo@meiragtx.com

Locations
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United States, Massachusetts
Massachusetts Eye and Ear Institute Recruiting
Boston, Massachusetts, United States, 02114
Contact: Rachel Huckfeldt, MD       Rachel_Huckfeldt@MEEI.HARVARD.EDU   
United States, Michigan
Kellogg Eye Center Recruiting
Ann Arbor, Michigan, United States, 48105
Contact: Cagri Besirli, MD       cbesirli@med.umich.edu   
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
Bionical-Emas Pharma Ltd
Investigators
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Principal Investigator: James Bainbridge, Prof University College, London
More Information
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Responsible Party: MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier: NCT03252847     History of Changes
Other Study ID Numbers: MGT009
First Posted: August 17, 2017    Key Record Dates
Last Update Posted: July 17, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by MeiraGTx UK II Ltd:
XLRP RPGR
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
 Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn