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History of Changes for Study: NCT03252847
Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) Retinitis Pigmentosa GTPase Regulator (RPGR)
Latest version (submitted October 25, 2022) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 August 16, 2017 None (earliest Version on record)
2 September 7, 2017 Study Status and Study Identification
3 December 15, 2017 Contacts/Locations and Study Status
4 July 15, 2019 Contacts/Locations, Sponsor/Collaborators, Study Status, Oversight, Arms and Interventions and Study Identification
5 January 31, 2020 Sponsor/Collaborators, Contacts/Locations, Study Design and Study Status
6 March 11, 2020 Recruitment Status, Arms and Interventions, Contacts/Locations, Study Status and Study Description
7 October 15, 2021 Study Status
8 December 15, 2021 Recruitment Status, Study Status and Study Design
9 October 25, 2022 Study Status
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Study NCT03252847
Submitted Date:  August 16, 2017 (v1)

Open or close this module Study Identification
Unique Protocol ID: MGT009
Brief Title: Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) Retinitis Pigmentosa GTPase Regulator (RPGR)
Official Title: An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
Secondary IDs:
Open or close this module Study Status
Record Verification: August 2017
Overall Status: Recruiting
Study Start: July 14, 2017
Primary Completion: November 2020 [Anticipated]
Study Completion: November 2020 [Anticipated]
First Submitted: August 1, 2017
First Submitted that
Met QC Criteria:
August 16, 2017
First Posted: August 17, 2017 [Actual]
Last Update Submitted that
Met QC Criteria:
August 16, 2017
Last Update Posted: August 17, 2017 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: MeiraGTx UK II Ltd
Responsible Party: Sponsor
Collaborators: Syne Qua Non Limited
EMAS Pharma
Open or close this module Oversight
U.S. FDA-regulated Drug: No
U.S. FDA-regulated Device: No
Data Monitoring: Yes
Open or close this module Study Description
Brief Summary: A clinical trial of AAV2 vector for patients with X-linked retinitis pigmentosa (XLRP)
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.
Open or close this module Conditions
Conditions: X-Linked Retinitis Pigmentosa
Keywords: XLRP RPGR
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 1/Phase 2
Interventional Study Model: Single Group Assignment
Biological
Number of Arms: 3
Masking: None (Open Label)
Allocation: Non-Randomized
Enrollment: 36 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: Low dose AAV-RPGR
Single, subretinal administration of low dose AAV-RPGR
Genetic: AAV-RPGR
Open label, non-randomised, dose-escalation intervention
Experimental: Intermediate dose AAV-RPGR
Single, subretinal administration of intermediate dose AAV-RPGR
Genetic: AAV-RPGR
Open label, non-randomised, dose-escalation intervention
Experimental: High dose AAV-RPGR
Single, subretinal administration of high dose AAV-RPGR
Genetic: AAV-RPGR
Open label, non-randomised, dose-escalation intervention
Open or close this module Outcome Measures
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
Open or close this module Eligibility
Minimum Age: 5 Years
Maximum Age:
Sex: Male
Gender Based:
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

Open or close this module Contacts/Locations
Central Contact Person: Julie Bakobaki, MSc
Telephone: +44 (0)20 3866 4320
Email: julie.bakobaki@meiragtx.com
Central Contact Backup: Praseeda Thaikalloor, MSc
Telephone: +44 (0)20 3866 4320
Email: praseeda.thaikalloor@meiragtx.com
Study Officials: James Bainbridge, Prof
Principal Investigator
University College, London
Locations: United Kingdom
Moorfields Eye Hospital NHS Foundation Trust
[Recruiting]
London, United Kingdom
Contact:Contact: Sophie Connor 02075662821 sophie.connor@moorfields.nhs.co.uk
Contact:Principal Investigator: James Bainbridge, Prof
Open or close this module IPDSharing
Plan to Share IPD: No
Open or close this module References
Citations:
Links:
Available IPD/Information:

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