We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

This study is currently recruiting participants.
Verified September 2017 by MeiraGTx UK II Ltd
Sponsor:
ClinicalTrials.gov Identifier:
NCT03252847
First Posted: August 17, 2017
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.
Collaborators:
Syne Qua Non Limited
Emas Pharma Ltd.
Information provided by (Responsible Party):
MeiraGTx UK II Ltd
  Purpose
A clinical trial of AAV2 vector for patients with X-linked retinitis pigmentosa (XLRP)

Condition Intervention Phase
X-Linked Retinitis Pigmentosa Genetic: AAV-RPGR Phase 1 Phase 2

Study Type: Interventional
Study Design: 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 (AAV2/5-hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)

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 sub retinal administration of AAV2-RPGR [ Time Frame: 18 months ]
    Safety is defined as the absence of ATIMP-related safety events


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

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

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


Estimated Enrollment: 36
Actual Study Start Date: July 14, 2017
Estimated Study Completion Date: November 2020
Estimated Primary Completion Date: November 2020 (Final data collection date for primary outcome measure)
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

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.
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   5 Years and older   (Child, Adult, Senior)
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
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
Contact: Julie Bakobaki, MSc +44 (0)20 3866 4320 julie.bakobaki@meiragtx.com
Contact: Praseeda Thaikalloor, MSc +44 (0)20 3866 4320 praseeda.thaikalloor@meiragtx.com

Locations
United Kingdom
Moorfields Eye Hospital NHS Foundation Trust Recruiting
London, United Kingdom
Contact: Sophie Connor    02075662821    sophie.connor@moorfields.nhs.co.uk   
Principal Investigator: James Bainbridge, Prof         
Sponsors and Collaborators
MeiraGTx UK II Ltd
Syne Qua Non Limited
Emas Pharma Ltd.
Investigators
Principal Investigator: James Bainbridge, Prof University College, London
  More Information

Responsible Party: MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier: NCT03252847     History of Changes
Other Study ID Numbers: MGT009
First Submitted: August 1, 2017
First Posted: August 17, 2017
Last Update Posted: September 11, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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

Keywords provided by MeiraGTx UK II Ltd:
XLRP RPGR

Additional relevant MeSH terms:
Retinitis
Retinitis Pigmentosa
Cone-Rod Dystrophies
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn