Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) Retinitis Pigmentosa GTPase Regulator (RPGR)
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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 :
Active, not recruiting
First Posted : August 17, 2017
Last Update Posted : March 13, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
X-Linked Retinitis Pigmentosa | Genetic: AAV2/5-RPGR | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 46 participants |
Allocation: | 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 |

Arm | Intervention/treatment |
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Experimental: Low dose AAV2/5-RPGR
Single, subretinal administration of low dose AAV2/5-RPGR
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Genetic: AAV2/5-RPGR
Open label, non-randomised, dose-escalation intervention followed by randomised dose confirmation against a control arm |
Experimental: Intermediate dose AAV2/5-RPGR
Single, subretinal administration of intermediate dose AAV2/5-RPGR
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Genetic: AAV2/5-RPGR
Open label, non-randomised, dose-escalation intervention followed by randomised dose confirmation against a control arm |
Experimental: High dose AAV2/5-RPGR
Single, subretinal administration of high dose AAV2/5-RPGR
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Genetic: AAV2/5-RPGR
Open label, non-randomised, dose-escalation intervention followed by randomised dose confirmation against a control arm |
- 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
- 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

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, Older Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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

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
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 |
Principal Investigator: | James Bainbridge, Prof | University College, London |
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: | March 13, 2020 |
Last Verified: | March 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
XLRP RPGR |
Retinitis Retinitis Pigmentosa Retinal Diseases Eye Diseases |
Eye Diseases, Hereditary Retinal Dystrophies Retinal Degeneration Genetic Diseases, Inborn |