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Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

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.
 
ClinicalTrials.gov Identifier: NCT04671433
Recruitment Status : Recruiting
First Posted : December 17, 2020
Last Update Posted : February 13, 2023
Sponsor:
Collaborator:
Janssen Research & Development, LLC
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Brief Summary:
A clinical trial of AAV5-RPGR vector for patients with X-linked retinitis pigmentosa (XLRP)

Condition or disease Intervention/treatment Phase
X-Linked Retinitis Pigmentosa Biological: Genetic: AAV5-RPGR Phase 3

Detailed Description:
Deferred Treatment

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 96 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Bilateral, subretinal administration of AAV5-RPGR - immediate treatment group
Masking: Single (Outcomes Assessor)
Masking Description: No intervention - deferred treatment group (Bilateral, subretinal administration of AAV5-RPGR to be administered in the follow-up study)
Primary Purpose: Treatment
Official Title: Phase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Actual Study Start Date : March 16, 2021
Estimated Primary Completion Date : March 2024
Estimated Study Completion Date : March 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Experimental - Immediate Treatment
RPGR4e11
Biological: Genetic: AAV5-RPGR
Bilateral, sub-retinal administration of AAV5-RPGR - immediate treatment group

Deferred Treatment
Deferred Treatment
Biological: Genetic: AAV5-RPGR
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-RPGR to be administered in the follow-up study)

Active Comparator: Experimental Immediate Treatment
RPGR2e11
Biological: Genetic: AAV5-RPGR
Bilateral, sub-retinal administration of AAV5-RPGR - immediate treatment group




Primary Outcome Measures :
  1. Change from baseline to Week 52 in vision-guided mobility assessment (VMA) as measured by the ability of the participant to navigate through a VMA maze [ Time Frame: 52 Weeks ]
    Change from baseline to Week 52 in vision-guided mobility assessment (VMA) as measured by the ability of the participant to navigate through a VMA maze


Secondary Outcome Measures :
  1. Change in retinal function as assessed by mean retinal sensitivity within the central 10 degrees (MRS10) in static perimetry at Week 52 [ Time Frame: Day 1 - Week 52 ]
    Mean retinal sensitivity within the central 10 degrees (MRS10) in static perimetry at Week 52

  2. Change in retinal function as assessed by pointwise responder in full visual field at Week 52 [ Time Frame: Day 1 - Week 52 ]
    Pointwise responder in full visual field at Week 52

  3. Change in retinal function as assessed by pointwise responder in the central 30 degrees visual field at Week 52 [ Time Frame: Day 1 - Week 52 ]
    Pointwise responder in the central 30 degrees visual field at Week 52

  4. Change in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52 [ Time Frame: Day 1 - Week 52 ]
    Mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52

  5. Change in functional vision as measured by patient-reported outcomes at Week 52 [ Time Frame: Day 1 - Week 52 ]
    Change in functional vision as measured by patient-reported outcomes at Week 52

  6. Change in visual function as assessed by binocular low luminance visual acuity using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score at Week 52 [ Time Frame: Day 1 - Week 52 ]
    Binocular low luminance visual acuity using the ETDRS chart letter score at Week 52

  7. Change in visual function as assessed by binocular best corrected visual acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score at Week 52 [ Time Frame: Day 1 - Week 52 ]
    Binocular BCVA using the ETDRS chart letter score at Week 52

  8. Safety and tolerability through Week 52 as assessed by: [ Time Frame: Day 1 - Week 52 ]
    • Number of participants with ocular and non-ocular adverse events
    • Number of participants with abnormalities in Hematologic and Clinical Chemistry parameters



Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female
  • 3 years of age or older
  • has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory.

Exclusion Criteria:

-


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): NCT04671433


Contacts
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Contact: MeiraGTx Clinical Project Manager +44 (0)20 3866 4320 info@lumeosstudy.com

Locations
Show Show 20 study locations
Sponsors and Collaborators
MeiraGTx UK II Ltd
Janssen Research & Development, LLC
Investigators
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Principal Investigator: James Bainbridge, MD Moorfield Eye Hospital (MEH)
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Responsible Party: MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier: NCT04671433    
Other Study ID Numbers: MGT-RPGR-021
First Posted: December 17, 2020    Key Record Dates
Last Update Posted: February 13, 2023
Last Verified: February 2023
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
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