Genetic: AAV5-RPGR 4e11 for Retinitis Pigmentosa

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Retinitis Pigmentosa+2 More
Genetic: AAV5-RPGR 4e11 - Biological
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a new vector (AAV5-RPGR) to see if it improves vision in patients with XLRP, a rare genetic disease that causes blindness.

Eligible Conditions
  • Retinitis Pigmentosa

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Other trials for Retinitis Pigmentosa

Study Objectives

1 Primary · 7 Secondary · Reporting Duration: Month 60

Month 60
Change from baseline in visual function as measured by Early Treatment of Diabetic Retinopathy Study (ETDRS)
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 abnormalities in Hematologic and Clinical Chemistry parameters
Safety and tolerability of bilateral treatment with AAV5-RPGR as measured by: Number of participants with ocular and non-ocular adverse events
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 functional vision Change from baseline in functional vision as measured by visual mobility assessment
Month 60
Change from baseline in retinal sensitivity as measured by perimetry
Through Month 60
To assess changes in visual function: Contrast sensitivity by Pelli-Robson chart in LogMAR over time through Month 60

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Retinitis Pigmentosa

Trial Design

3 Treatment Groups

Already treated in MGT-RPGR-021
1 of 3
Deferred treatment from MGT-RPGR-021 4e11
1 of 3
Deferred treatment from MGT-RPGR-021 2e11
1 of 3
Active Control
Experimental Treatment

66 Total Participants · 3 Treatment Groups

Primary Treatment: Genetic: AAV5-RPGR 4e11 · No Placebo Group · Phase 3

Deferred treatment from MGT-RPGR-021 4e11
Biological
Experimental Group · 1 Intervention: Genetic: AAV5-RPGR 4e11 · Intervention Types: Biological
Deferred treatment from MGT-RPGR-021 2e11
Biological
Experimental Group · 1 Intervention: Genetic: AAV5-RPGR 2e11 · Intervention Types: Biological
Already treated in MGT-RPGR-021ActiveComparator Group · 2 Interventions: Genetic: AAV5-RPGR 4e11, Genetic: AAV5-RPGR 2e11 · Intervention Types: Biological, Biological

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: month 60

Who is running the clinical trial?

MeiraGTx UK II LtdLead Sponsor
14 Previous Clinical Trials
891 Total Patients Enrolled
4 Trials studying Retinitis Pigmentosa
401 Patients Enrolled for Retinitis Pigmentosa
Janssen Research & Development, LLCIndustry Sponsor
918 Previous Clinical Trials
6,327,100 Total Patients Enrolled
2 Trials studying Retinitis Pigmentosa
67 Patients Enrolled for Retinitis Pigmentosa
James Bainbridge, MDPrincipal InvestigatorMoorfield Eye Hospital (MEH)
1 Previous Clinical Trials
66 Total Patients Enrolled
1 Trials studying Retinitis Pigmentosa
66 Patients Enrolled for Retinitis Pigmentosa

Eligibility Criteria

Age Any Age · All Participants · 1 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 21st, 2021

Last Reviewed: October 11th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

Who else is applying?

What state do they live in?
Ohio28.6%
California28.6%
Texas14.3%
Other28.6%
How old are they?
< 1850.0%
18 - 6550.0%
What site did they apply to?
Boston Eye and Ear40.0%
Retinal Consultants of Houston20.0%
Children's Hospital Los Angeles40.0%
What portion of applicants met pre-screening criteria?
Met criteria100.0%

How responsive is this trial?

Most responsive sites:
  1. Children's Hospital Los Angeles: < 24 hours
  2. Boston Eye and Ear: < 48 hours
Average response time
  • < 2 Days
Typically responds via
Phone Call33.3%
Email66.7%