Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Primary Purpose
X-Linked Retinitis Pigmentosa
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Genetic: AAV5-RPGR
Genetic: AAV5-RPGR
Sponsored by
About this trial
This is an interventional treatment trial for X-Linked Retinitis Pigmentosa
Eligibility 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:
-
Sites / Locations
- Children's Hospital Los Angeles
- Stanford University
- Shiley Eye Institute, University of California San Diego
- Vitreo Retinal Associates
- The Emory Eye Centre
- Massachusetts Eye and Ear, Centre for Clinical Research Operations
- University of Michigan, W.K. Kellogg Eye Centre
- Duke Eye Center
- University of Pittsburgh Medical Centre
- Retinal Consultants of Houston
- Ghent University Hospital
- The Hospital for Sick Children
- Rigshospitalet Glostrup
- Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts
- Hadassah Medical Center
- Azienda Ospedaliero, Universitaria Careggi
- Ospedale San Paolo di Milano
- AOU Università degli, Studi della Campania
- IRCCS Fondazione G. B. Bietti , Ospedale Britannico
- Amsterdam UMC
- Radboud University Medical Center
- University Hospital Fundación Jiménez Díaz
- University Hospital Basel
- Hôpital ophtalmique Jules-Gonin
- NHS Lothian
- Glasgow Centre for Ophthalmic Research, Gartnaval General Hospital
- Leeds Centre for Ophthalmology, St James' University Hospital
- Moorfield Eye Hospital (MEH)
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Other
Active Comparator
Arm Label
Experimental - Immediate Treatment
Deferred Treatment
Experimental Immediate Treatment
Arm Description
RPGR4e11
Deferred Treatment
RPGR2e11
Outcomes
Primary Outcome Measures
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
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
Change in retinal function as assessed by mean retinal sensitivity within the central 10 degrees (MRS10) in static perimetry at Week 52
Mean retinal sensitivity within the central 10 degrees (MRS10) in static perimetry at Week 52
Change in retinal function as assessed by pointwise responder in full visual field at Week 52
Pointwise responder in full visual field at Week 52
Change in retinal function as assessed by pointwise responder in the central 30 degrees visual field at Week 52
Pointwise responder in the central 30 degrees visual field at Week 52
Change in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52
Mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52
Change in functional vision as measured by patient-reported outcomes at Week 52
Change in functional vision as measured by patient-reported outcomes at Week 52
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
Binocular low luminance visual acuity using the ETDRS chart letter score at Week 52
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
Binocular BCVA using the ETDRS chart letter score at Week 52
Safety and tolerability through Week 52 as assessed by:
Number of participants with ocular and non-ocular adverse events
Number of participants with abnormalities in Hematologic and Clinical Chemistry parameters
Full Information
NCT ID
NCT04671433
First Posted
November 5, 2020
Last Updated
September 21, 2023
Sponsor
MeiraGTx UK II Ltd
Collaborators
Janssen Research & Development, LLC
1. Study Identification
Unique Protocol Identification Number
NCT04671433
Brief Title
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
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
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
March 16, 2021 (Actual)
Primary Completion Date
September 2024 (Anticipated)
Study Completion Date
September 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MeiraGTx UK II Ltd
Collaborators
Janssen Research & Development, LLC
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
A clinical trial of AAV5-RPGR vector for patients with X-linked retinitis pigmentosa (XLRP)
Detailed Description
Deferred Treatment
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
X-Linked Retinitis Pigmentosa
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Model Description
Bilateral, subretinal administration of AAV5-RPGR - immediate treatment group
Masking
Outcomes Assessor
Masking Description
No intervention - deferred treatment group (Bilateral, subretinal administration of AAV5-RPGR to be administered in the follow-up study)
Allocation
Randomized
Enrollment
97 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Experimental - Immediate Treatment
Arm Type
Experimental
Arm Description
RPGR4e11
Arm Title
Deferred Treatment
Arm Type
Other
Arm Description
Deferred Treatment
Arm Title
Experimental Immediate Treatment
Arm Type
Active Comparator
Arm Description
RPGR2e11
Intervention Type
Biological
Intervention Name(s)
Genetic: AAV5-RPGR
Intervention Description
Bilateral, sub-retinal administration of AAV5-RPGR - immediate treatment group
Intervention Type
Biological
Intervention Name(s)
Genetic: AAV5-RPGR
Intervention Description
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-RPGR to be administered in the follow-up study)
Primary Outcome Measure Information:
Title
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
Description
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
Secondary Outcome Measure Information:
Title
Change in retinal function as assessed by mean retinal sensitivity within the central 10 degrees (MRS10) in static perimetry at Week 52
Description
Mean retinal sensitivity within the central 10 degrees (MRS10) in static perimetry at Week 52
Time Frame
Day 1 - Week 52
Title
Change in retinal function as assessed by pointwise responder in full visual field at Week 52
Description
Pointwise responder in full visual field at Week 52
Time Frame
Day 1 - Week 52
Title
Change in retinal function as assessed by pointwise responder in the central 30 degrees visual field at Week 52
Description
Pointwise responder in the central 30 degrees visual field at Week 52
Time Frame
Day 1 - Week 52
Title
Change in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52
Description
Mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52
Time Frame
Day 1 - Week 52
Title
Change in functional vision as measured by patient-reported outcomes at Week 52
Description
Change in functional vision as measured by patient-reported outcomes at Week 52
Time Frame
Day 1 - Week 52
Title
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
Description
Binocular low luminance visual acuity using the ETDRS chart letter score at Week 52
Time Frame
Day 1 - Week 52
Title
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
Description
Binocular BCVA using the ETDRS chart letter score at Week 52
Time Frame
Day 1 - Week 52
Title
Safety and tolerability through Week 52 as assessed by:
Description
Number of participants with ocular and non-ocular adverse events
Number of participants with abnormalities in Hematologic and Clinical Chemistry parameters
Time Frame
Day 1 - Week 52
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility 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:
-
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
James Bainbridge, MD
Organizational Affiliation
Moorfield Eye Hospital (MEH)
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94303
Country
United States
Facility Name
Shiley Eye Institute, University of California San Diego
City
San Diego
State/Province
California
ZIP/Postal Code
92093-0946
Country
United States
Facility Name
Vitreo Retinal Associates
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32607
Country
United States
Facility Name
The Emory Eye Centre
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Massachusetts Eye and Ear, Centre for Clinical Research Operations
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
University of Michigan, W.K. Kellogg Eye Centre
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States
Facility Name
Duke Eye Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
22710
Country
United States
Facility Name
University of Pittsburgh Medical Centre
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Retinal Consultants of Houston
City
Bellaire
State/Province
Texas
ZIP/Postal Code
77401
Country
United States
Facility Name
Ghent University Hospital
City
Ghent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
The Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
Rigshospitalet Glostrup
City
Glostrup
ZIP/Postal Code
2600
Country
Denmark
Facility Name
Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts
City
Paris
ZIP/Postal Code
75012
Country
France
Facility Name
Hadassah Medical Center
City
Jerusalem
ZIP/Postal Code
91120
Country
Israel
Facility Name
Azienda Ospedaliero, Universitaria Careggi
City
Florence
Country
Italy
Facility Name
Ospedale San Paolo di Milano
City
Milan
Country
Italy
Facility Name
AOU Università degli, Studi della Campania
City
Napoli
Country
Italy
Facility Name
IRCCS Fondazione G. B. Bietti , Ospedale Britannico
City
Roma
Country
Italy
Facility Name
Amsterdam UMC
City
Amsterdam
ZIP/Postal Code
1105AZ
Country
Netherlands
Facility Name
Radboud University Medical Center
City
Nijmegen
ZIP/Postal Code
6525EX
Country
Netherlands
Facility Name
University Hospital Fundación Jiménez Díaz
City
Madrid
ZIP/Postal Code
28040
Country
Spain
Facility Name
University Hospital Basel
City
Basel
ZIP/Postal Code
4031
Country
Switzerland
Facility Name
Hôpital ophtalmique Jules-Gonin
City
Lausanne
ZIP/Postal Code
1004
Country
Switzerland
Facility Name
NHS Lothian
City
Edinburgh
ZIP/Postal Code
EH3 9HA
Country
United Kingdom
Facility Name
Glasgow Centre for Ophthalmic Research, Gartnaval General Hospital
City
Glasgow
ZIP/Postal Code
G12 0YN
Country
United Kingdom
Facility Name
Leeds Centre for Ophthalmology, St James' University Hospital
City
Leeds
ZIP/Postal Code
LS9 7TF
Country
United Kingdom
Facility Name
Moorfield Eye Hospital (MEH)
City
London
ZIP/Postal Code
EC1V 2PD
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
We'll reach out to this number within 24 hrs