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Virus Therapy

Gene Therapy for Retinitis Pigmentosa

Phase 3
Waitlist Available
Led By James Bainbridge, MD
Research Sponsored by MeiraGTx UK II Ltd
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 1 - week 52
Awards & highlights

Study Summary

This trial is testing a new vector for treating XLRP, a disease that causes blindness.

Who is the study for?
This trial is for males and females aged 3 or older with X-linked retinitis pigmentosa (XLRP) confirmed by a specialist. Participants must have a specific genetic variant in the RPGR gene verified by an accredited lab.Check my eligibility
What is being tested?
The trial is testing AAV5-RPGR, a form of gene therapy designed to treat XLRP. This intervention involves using a harmless virus to deliver corrected genes into the body.See study design
What are the potential side effects?
Potential side effects of AAV5-RPGR may include immune reactions, eye inflammation or discomfort, changes in vision, headache, and possible mild fever post-treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am at least 3 years old.
Select...
I have X-linked retinitis pigmentosa confirmed by a specialist and genetic testing.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1 - week 52
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 - week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
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
Secondary outcome measures
Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52
Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52
Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52
+11 more

Trial Design

3Treatment groups
Experimental Treatment
Group I: Experimental Immediate TreatmentExperimental Treatment1 Intervention
Low dose.
Group II: Experimental - Immediate TreatmentExperimental Treatment1 Intervention
Intermediate dose.
Group III: Deferred TreatmentExperimental Treatment1 Intervention
Deferred Treatment

Find a Location

Who is running the clinical trial?

MeiraGTx UK II LtdLead Sponsor
14 Previous Clinical Trials
626 Total Patients Enrolled
4 Trials studying Retinitis Pigmentosa
341 Patients Enrolled for Retinitis Pigmentosa
Janssen Research & Development, LLCLead Sponsor
975 Previous Clinical Trials
6,384,211 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
154 Patients Enrolled for Retinitis Pigmentosa
Janssen Research & Development, LLC Clinical TrialStudy DirectorJanssen Research & Development, LLC
745 Previous Clinical Trials
3,959,808 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
154 Patients Enrolled for Retinitis Pigmentosa

Media Library

AAV5-RPGR (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04671433 — Phase 3
Retinitis Pigmentosa Research Study Groups: Deferred Treatment, Experimental - Immediate Treatment, Experimental Immediate Treatment
Retinitis Pigmentosa Clinical Trial 2023: AAV5-RPGR Highlights & Side Effects. Trial Name: NCT04671433 — Phase 3
AAV5-RPGR (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04671433 — Phase 3
Retinitis Pigmentosa Patient Testimony for trial: Trial Name: NCT04671433 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are new patients being accepted for this program at this time?

"The most up-to-date information on clinicaltrials.gov suggests that this trial is still recruiting patients. This particular clinical trial was first posted on March 16th, 2021 and was last edited on September 22nd, 2022."

Answered by AI

Who else is applying?

What state do they live in?
Puerto Rico
California
Texas
Other
How old are they?
18 - 65
What site did they apply to?
The Emory Eye Centre
Retinal Consultants of Houston
Aaron Los Nagiel
Other
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
0

What questions have other patients asked about this trial?

How long is each trail?
PatientReceived no prior treatments

Why did patients apply to this trial?

I was diagnosed with RP in Jan 2019 mutation EYS c1867del,p(Leu623Phefs*20)and EYSc.3562C>T,p(Gln1188*).
PatientReceived no prior treatments
Im applying for this trial to save some of my vision. What motivates me is that there's hope for me my mom and others .
PatientReceived no prior treatments

How responsive is this trial?

Typically responds via
Phone Call
Most responsive sites:
  1. Retinal Consultants of Houston: < 48 hours
Average response time
  • < 2 Days
Recent research and studies
clinicaltrials.govStudy
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
2020. "Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04671433.
All > Retinitis Pigmentosa Boston > Retinitis Pigmentosa
~9 spots leftby Sep 2024
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