Gene Therapy for Retinitis Pigmentosa
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a gene therapy for people with X-linked retinitis pigmentosa. It uses a harmless virus to deliver a healthy gene to eye cells, aiming to improve their function and slow down vision loss. Gene therapy targeting the RPE65 gene has shown promise in treating inherited retinal dystrophies, including retinitis pigmentosa.
Do I need to stop my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications.
Is gene therapy for retinitis pigmentosa safe for humans?
How is the AAV5-RPGR treatment different from other treatments for retinitis pigmentosa?
AAV5-RPGR is a gene therapy that uses a modified virus to deliver a healthy copy of the RPGR gene directly to the eye's photoreceptor cells, which is different from other treatments that may not target the genetic cause of the disease. This approach is unique because it aims to correct the underlying genetic defect, potentially preserving or improving vision in patients with retinitis pigmentosa caused by RPGR mutations.24567
What data supports the effectiveness of the treatment AAV5-RPGR for retinitis pigmentosa?
Gene therapy using similar treatments has shown promise in improving vision in patients with retinitis pigmentosa, with some studies reporting visual field improvements and preserved photoreceptor function in animal models. Additionally, early human trials have shown safety and some visual improvements, suggesting potential effectiveness.12348
Who Is on the Research Team?
Janssen Research & Development, LLC Clinical Trial
Principal Investigator
Janssen Research & Development, LLC
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive the AAV5-hRKp.RPGR vector for the treatment of X-linked retinitis pigmentosa
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- AAV5-RPGR
Find a Clinic Near You
Who Is Running the Clinical Trial?
MeiraGTx UK II Ltd
Lead Sponsor
Janssen Research & Development, LLC
Lead Sponsor
Joaquin Duato
Janssen Research & Development, LLC
Chief Executive Officer since 2022
MBA from ESADE, Master of International Management from Thunderbird School of Global Management
Dr. Jijo James, MD
Janssen Research & Development, LLC
Chief Medical Officer since 2014
MD from St. Johns Medical College, MPH from Columbia University