Gene Therapy for Retinitis Pigmentosa
(RESTORE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new eye injection that uses a virus to deliver a helpful protein to improve vision in patients with severe vision loss from Advanced Retinitis Pigmentosa.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is gene therapy for retinitis pigmentosa safe for humans?
In a study on gene therapy for X-linked retinitis pigmentosa, the treatment was generally safe, with only some patients experiencing inflammation that responded to steroids. Another study in dogs showed that a similar gene therapy did not cause inflammation or immune reactions, suggesting it is safe.12345
What makes the treatment MCO-010 for retinitis pigmentosa unique?
The treatment MCO-010 for retinitis pigmentosa is unique because it involves gene therapy, which aims to address the genetic cause of the disease by delivering a healthy copy of the gene directly to the retina, potentially offering a more targeted and long-lasting solution compared to traditional treatments that only manage symptoms.25678
What data supports the effectiveness of the treatment MCO-010 for retinitis pigmentosa?
Research shows that using multicharacteristic opsin (MCO) in animal models with retinal degeneration led to significant vision improvement. The treatment was delivered safely to specific retinal cells and improved visually guided behavior, suggesting potential effectiveness for retinitis pigmentosa.3591011
Who Is on the Research Team?
Dr Samarendra Mohanty
Principal Investigator
Nanoscope Therapeutics Inc.
Are You a Good Fit for This Trial?
Adults over 18 with advanced Retinitis Pigmentosa (RP) and very poor vision can join this trial. They must have a confirmed diagnosis of RP, some remaining retinal layers visible on OCT scans, and understand the study to give consent. Excluded are those with other eye diseases like glaucoma, recent eye surgery, or infections like HIV; those who've had gene therapy before; or cannot perform mobility tests.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intravitreal injection of MCO-010 or sham injection
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- MCO-010
- Sham Injection
- vMCO-010
Find a Clinic Near You
Who Is Running the Clinical Trial?
Nanoscope Therapeutics Inc.
Lead Sponsor