SLO-RP Preserve is a global clinical trial evaluating whether NPI-001 (N-acetylcysteine amide) tablets can slow vision loss caused by Retinitis Pigmentosa (RP) in patients with Usher Syndrome (USH).
In this clinical trial, participants will receive either NPI-001 tablets or a placebo. A placebo looks like the trial drug but has no active ingredient. Comparing the two helps researchers learn if NPI-001 tablets can slow vision loss and improve quality of life.
SLO-RP Preserve is a confirmatory Phase 3 clinical trial studying whether investigational NPI-001 oral tablets can slow vision loss caused by RP associated with Usher Syndrome. This trial is not designed to treat or assess hearing loss associated with Usher Syndrome.
Participation in the study will last approximately two years. This includes a screening visit, a baseline visit and seven follow-up study visits. Twenty-four months of treatment is planned for all trial participants.
If you qualify and join the study, you will receive oral NPI-001 or placebo tablets. You will take one tablet twice a day, one in the morning and one in the evening. NPI-001 is an investigational drug that has not been previously approved in any country.
SLO-RP Preserve is enrolling participants at sites in the United States and Australia.
VIEW SITESUsher Syndrome (USH) is an inherited condition that affects both hearing and vision. The vision loss of USH is due to Retinitis Pigmentosa (RP), which is a group of eye diseases that gradually damage the retina, the light-sensitive tissue at the back of the eye.
As the retina becomes damaged, people may have trouble seeing at night and can slowly lose their peripheral or side vision. Symptoms often begin during adolescence or early adulthood. Because USH affects people differently, each person’s experience can be unique.
NPI-001 is an investigational oral treatment being studied for retinitis pigmentosa (RP), a group of inherited retinal diseases that can cause progressive vision loss.
NPI-001 is designed to help reduce oxidative stress, an imbalance that may happen in the back of the eye in people with RP. This imbalance can lead to cell damage, contributing to vision loss in people with RP over time. In previous clinical studies, NPI-001 helped protect retinal cells from damage, slowing photoreceptor loss.
NPI-001 is being developed as a tablet that can be taken by mouth and administered at home.
The U.S. Food and Drug Administration (FDA) has granted NPI-001 Breakthrough Therapy, Fast Track and Orphan Drug designations for the treatment of RP. These designations are intended to help support the development and review of potential treatments for serious or rare diseases.
If you qualify for this study, here is what you can expect:
Study Visits: You will be asked to attend nine in-clinic visits over approximately two years.
Study Drug: The study drug is in the form of a tablet and will be given to you to self-administer twice a day – one tablet in the morning and one tablet in the evening. This study also includes a placebo. Therefore, there is a chance (50/50) that you may be assigned to the placebo group during the study. The study doctor and staff will not know which study drug you are assigned, unless medically necessary.
Compensation: You may be eligible to receive compensation for your time and participation.
During each study visit, your study team will guide you through a series of assessments to monitor your health and the study treatment. These assessments may include:
You will be asked about your overall health, past medical conditions, medications and previous eye treatments.
Various eye examinations to assess your eye health.
These samples will be collected for safety reasons to monitor your health while taking the study medication.
You will complete simple surveys about your eye symptoms and experiences.