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Gene 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
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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline- month 60
Awards & highlights

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.

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 change in the RPGR gene verified by an accredited lab.Check my eligibility
What is being tested?
The study tests two doses of AAV5-RPGR, a gene therapy vector, to treat XLRP. Patients will receive one of the two doses to see how well it works and what side effects occur.See study design
What are the potential side effects?
Potential side effects are not specified here but may include typical reactions related to gene therapy such as immune response, eye irritation or inflammation, and vision changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am 3 or older with XLRP confirmed by a specialist and a genetic test.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline- month 60
This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline- month 60 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 in vision-guided mobility assessment (VMA), as measured by the ability of the participant to navigate through a VMA maze, after bilateral subretinal delivery of AAV5-hRKp.RPGR
Number of Participants With Abnormalities in Laboratory Assessments
Number of participants with Ocular and Non-ocular Adverse Events of AAV5-hRKp.RPGR in Participants with RPGR-XLRP
Secondary outcome measures
Change From Baseline in Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Score in Monocular Assessment
Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Worse-seeing Eye
Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment
+9 more

Trial Design

3Treatment groups
Experimental Treatment
Group I: Deferred Treatment From MGT-RPGR-021 of Intermediate DoseExperimental Treatment1 Intervention
Deferred treatment
Group II: Deferred Treatment From MGT-RPGR-021 Low DoseExperimental Treatment1 Intervention
Deferred treatment
Group III: Already Treated in MGT-RPGR-021Experimental Treatment2 Interventions
Already treated

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 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04794101 — Phase 3
Retinitis Pigmentosa Research Study Groups: Deferred Treatment From MGT-RPGR-021 Low Dose, Deferred Treatment From MGT-RPGR-021 of Intermediate Dose, Already Treated in MGT-RPGR-021
Retinitis Pigmentosa Clinical Trial 2023: AAV5-RPGR Highlights & Side Effects. Trial Name: NCT04794101 — Phase 3
AAV5-RPGR (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04794101 — Phase 3
Retinitis Pigmentosa Patient Testimony for trial: Trial Name: NCT04794101 — Phase 3

Frequently Asked Questions

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

Are there any dangers to patients when they receive the gene therapy AAV5-RPGR 4e11?

"Genetic: AAV5-RPGR 4e11 is in Phase 3 clinical trials, meaning that there is evidence of efficacy and multiple rounds of data supporting safety. Therefore, it received a score of 3 for safety."

Answered by AI

If a patient wanted to participate in this trial, how many different places would they have to go?

"There are a total of 8 sites running this trial. They are located in Boston, Atlanta, Los Angeles as well other cities. If you enroll and have to travel for the trial, it would be best to choose the site nearest you to cut down on time and effort commuting."

Answered by AI

Who else is applying?

What state do they live in?
Ohio
Texas
California
Other
How old are they?
< 18
18 - 65
What site did they apply to?
Boston Eye and Ear
Retinal Consultants of Houston
Children's Hospital Los Angeles
Other
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
2
0

Why did patients apply to this trial?

I have grandkids to raise. I’ll do anything at this point 🙏🏼. I am apply for this trial because there is hope that I can stop the process of loosing my vision. What motivates me is that I can be a part of of a cure for me my mom and 1,000 of people.
PatientReceived 1 prior treatment

How responsive is this trial?

Average response time
  • < 2 Days
Most responsive sites:
  1. Children's Hospital Los Angeles: < 24 hours
  2. Boston Eye and Ear: < 48 hours
Typically responds via
Phone Call
Email
Recent research and studies
clinicaltrials.govStudy
Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
2020. "Follow-up 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/NCT04794101.
All > Retinitis Pigmentosa Boston > Retinitis Pigmentosa
~60 spots leftby Sep 2029
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