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

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: NCT03252847
Recruitment Status : Completed
First Posted : August 17, 2017
Last Update Posted : December 7, 2022
Sponsor:
Collaborators:
Syne Qua Non Limited
Bionical Emas
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Study Description
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Brief Summary:

The Phase 1 part of the study is a dose escalation of subretinal administration of AAV2/5 vector to assess the safety of this vector in participants with XLRP caused by mutations in RPGR.

The Phase 2 part of the study is a cohort expansion of subretinal administration of AAV2/5 vector to assess the safety and efficacy of this vector in participants with XLRP caused by mutations in RPGR.


Condition or disease Intervention/treatment Phase
X-Linked Retinitis Pigmentosa Genetic: AAV2/5-RPGR Phase 1 Phase 2

Detailed Description:
This is an open-label phase 1/2 dose-escalation and cohort expansion trial to determine the safety and efficacy of subretinal administration of AAV2/5 vector in participants with XLRP caused by mutations in RPGR.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 49 participants
Allocation: Randomized
Intervention Model: Sequential 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
Actual Primary Completion Date : November 18, 2021
Actual Study Completion Date : November 18, 2021

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Phase 1 (Part 1, Dose Escalation)
Participants receive one of three doses of AAV2/5-RPGR
 Genetic: AAV2/5-RPGR
Single, subretinal administration of AAV2/5-RPGR
Experimental: Phase 2 (Part 2; Expansion)
Participants receive one of two doses of AAV2/5-RPGR
 Genetic: AAV2/5-RPGR
Single, subretinal administration of AAV2/5-RPGR


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 advanced therapy medicinal product (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


Locations
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United States, Massachusetts
Massachusetts Eye and Ear Institute
Boston, Massachusetts, United States, 02114
United States, Michigan
Kellogg Eye Center
Ann Arbor, Michigan, United States, 48105
United States, Pennsylvania
UPMC Eye Center
Pittsburgh, Pennsylvania, United States, 15213
United Kingdom
Leeds Teaching Hospitals NHS Trust
Leeds, United Kingdom, LS9 7TF
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom
Sponsors and Collaborators
MeiraGTx UK II Ltd
Syne Qua Non Limited
Bionical Emas
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    
Other Study ID Numbers: MGT009
First Posted: August 17, 2017    Key Record Dates
Last Update Posted: December 7, 2022
Last Verified: November 2022
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