Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Sponsor
MeiraGTx UK II Ltd (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04794101
Collaborator
Janssen Research & Development, LLC (Industry)
66
10
3
90.5
6.6
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Study Details
Study Description
Brief Summary
A clinical trial of AAV5-RPGR vector for patients with X-linked retinitis pigmentosa (XLRP)
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Study Design
Study Type:
Interventional
Anticipated Enrollment
:
66 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Intervention Model Description:
66 Patients randomised in MGT-RPGR-021. 44 patients randomised to Immediate treatment arm. 22 patients randomised to the deferred arm. These patients will be treated in MGT-RPGR-022. All patients to be followed for 60 months.66 Patients randomised in MGT-RPGR-021. 44 patients randomised to Immediate treatment arm. 22 patients randomised to the deferred arm. These patients will be treated in MGT-RPGR-022. All patients to be followed for 60 months.
Masking:
Single (Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
Follow-up 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
:
Mar 16, 2021
Anticipated Primary Completion Date
:
Oct 1, 2024
Anticipated Study Completion Date
:
Oct 1, 2028
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Deferred treatment from MGT-RPGR-021 4e11 Deferred treatment |
Biological: Genetic: AAV5-RPGR 4e11
Bilateral, subretinal administration of AAV5-RPGR - deferred treatment group
|
| Active Comparator: Already treated in MGT-RPGR-021 Already treated |
Biological: Genetic: AAV5-RPGR 4e11
Bilateral, subretinal administration of AAV5-RPGR - deferred treatment group
Biological: Genetic: AAV5-RPGR 2e11
Bilateral, subretinal administration of AAV5-RPGR - deferred treatment group
|
| Experimental: Deferred treatment from MGT-RPGR-021 2e11 Deferred treatment |
Biological: Genetic: AAV5-RPGR 2e11
Bilateral, subretinal administration of AAV5-RPGR - deferred treatment group
|
Outcome Measures
Primary Outcome Measures
- Change from baseline in retinal sensitivity as measured by perimetry [Month 60]
Change from baseline in retinal sensitivity as measured by perimetry
Secondary Outcome Measures
- Change from baseline in visual function as measured by Early Treatment of Diabetic Retinopathy Study (ETDRS) [Day 0 - Month 60]
Change from baseline in visual function as measured by Early Treatment of Diabetic Retinopathy Study (ETDRS)
- To assess changes in functional vision Change from baseline in functional vision as measured by visual mobility assessment [Day 0 - Month 60]
To assess changes in functional vision Change from baseline in functional vision as measured by visual mobility assessment
- To assess changes from baseline in patient-reported functional vision as assessed by the patient-reported outcome questionnaire, Impact of Vision Impairment (IVI) - 'Reading and Accessing Information' domain [Day 0 - Month 60]
To assess changes from baseline in patient-reported functional vision as assessed by the patient-reported outcome questionnaire, Impact of Vision Impairment (IVI) - 'Reading and Accessing Information' domain
- To assess changes in visual function: Contrast sensitivity by Pelli-Robson chart in LogMAR over time through Month 60 [Through Month 60]
Contrast sensitivity by Pelli-Robson chart in LogMAR over time through Month 60
- Change from baseline in visual function as measured by contrast sensitivity [Day 0 - Month 60]
Change from baseline in visual function as measured by contrast sensitivity
- Safety and tolerability of bilateral treatment with AAV5-RPGR as measured by: Number of participants with ocular and non-ocular adverse events [Day 0 - Month 60]
Number of participants with ocular and non-ocular adverse events
- Safety and tolerability of bilateral treatment with AAV5-RPGR as measured by: Number of participants with abnormalities in Hematologic and Clinical Chemistry parameters [Day 0 - Month 60]
Number of participants with abnormalities in Hematologic and Clinical Chemistry parameters
Eligibility Criteria
Criteria
Ages Eligible for Study:
3 Years
and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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:
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Children's Hospital Los Angeles | Los Angeles | California | United States | 90027 |
| 2 | Vitreo Retinal Associates | Gainesville | Florida | United States | 32607 |
| 3 | The Emory Eye Centre | Atlanta | Georgia | United States | 30322 |
| 4 | Boston Eye and Ear | Boston | Massachusetts | United States | 02114 |
| 5 | Univeristy of Michigan | Ann Arbor | Michigan | United States | 48105 |
| 6 | Univeristy of Pittsburgh Medical Centre | Pittsburgh | Pennsylvania | United States | 15213 |
| 7 | Retinal Consultants of Houston | Bellaire | Texas | United States | 77401 |
| 8 | Hadassah Medical Center | Jerusalem | Israel | 91120 | |
| 9 | Amsterdam UMC | Amsterdam | Netherlands | 1105AZ | |
| 10 | Moorfield Eye Hospital (MEH) | London | United Kingdom | EC1V 2PD |
Sponsors and Collaborators
- MeiraGTx UK II Ltd
- Janssen Research & Development, LLC
Investigators
- Principal Investigator: James Bainbridge, MD, Moorfield Eye Hospital (MEH)
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.Responsible Party:
MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier:
NCT04794101
Other Study ID Numbers:
- MGT-RPGR-022
First Posted:
Mar 11, 2021
Last Update Posted:
Aug 17, 2022
Last Verified:
Aug 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms: