NPI-001 to protect eye cells in retinitis pigmentosa linked to Usher syndrome
Tests NPI-001 against placebo in adults with Usher syndrome and retinitis pigmentosa.
Plain-language title generated from the registry record
Registry title: A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
Key facts
- Where
- Dallas, United StatesSee the site
- Phase
- Phase 3
Where in United States
- Retina Foundation of the SouthwestDallas, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Nacuity Pharmaceuticals, Inc.
- Trial code
- NCT07710196
- Record updated
- 10 November 2026
- Original description
The goal of this clinical trial is to learn if NPI-001 works to prevent progression of retinitis pigmentosa in adults diagnosed with Usher syndrome. It will also provide information about the safety of NPI-001. The main questions it aims to answer are:Read full original descriptionShow less
A 24-Month, Randomized, Double-Masked, Placebo-Controlled Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis PigmentosaAssociated With Usher Syndrome
The goal of this clinical trial is to learn if NPI-001 works to prevent progression of retinitis pigmentosa in adults diagnosed with Usher syndrome. It will also provide information about the safety of NPI-001. The main questions it aims to answer are:
Does NPI-001 slow down the loss of photoreceptors? What medical problems do participants have when taking NPI-001? Researchers will compare NPI-001 to a placebo (a look-alike substance that contains no drug) to see if NPI-001 works to preserve vision.
Participants will:
Take NPI-001 or a placebo twice a day, every day for 24 months and visit the clinic 9 times for checkups and tests.
Inclusion: Able to comprehend and willing to sign an informed consent form (OCF) and adhere to the study protocol; Diagnosed with RP associated with Usher syndrome; EZ width ≥ 500 microns, which includes the fovea in each eye at Visit 1; All edges of the EZ area in both eyes can be visualized at Visit 1 (Screening); Have at least 20 detectable points on the MAIA grid in at least one eye at the Screening and Baseline visits (same eye for both visits); On stable dose of medications associated with other conditions for at least one month; Willing to use contraception during study (for anyone of childbearing potential).
Exclusion: Concurrent retinal pathologies that result in vision loss or inability to fixate; Intraocular surgery within the last two months or capsulotomy within the last month; Likely to require cataract surgery within the next 12 months; Use of any other investigational new drug, or participation in another clinical trial within 12 weeks before the start of study treatment; Use of N-acetylcysteine containing products in the previous 30 days prior to the baseline visit or unwilling to refrain from such supplements for the duration of the study; Current or history of uveitis, Coat's disease, diabetic retinopathy, glaucoma, herpes simplex of the eye, or currently has a cataract that prevents visualization of the posterior pole; Chronic liver or kidney disease, cystic fibrosis, severe asthma, or chronic obstructive pulmonary disease (COPD), history of thrombocytopenia not due to a reversible cause, or other blood dyscrasia; History of known sensitivity to N-acetylcysteine or similar thiol compounds or any ingredients of NPI-001; History of hypersensitivity to any medication or food resulting in systemic symptoms; History of cancer (other than non-melanoma skin cancer) diagnosed or requiring treatment within the past 2 years; Suspected liver dysfunction determined by having alanine aminotransferase (ALT), aspartate aminotransferase (AST), or bilirubin values > 1.5 X the upper limit of normal (ULN) at screening; Platelet or hemoglobin values < 100 at screening; Subjects who are unwilling or unable to refrain from donation of blood from 30 days prior to Screening, plasma from 2 weeks prior to Screening, or platelets from 6 weeks prior to Screening until 56 days following the last study drug dose administered; Unstable fixation during microperimetry in either eye at either Screening or Baseline visits; Pregnant women or women planning to become pregnant in the next 25 months or men with partners planning to become pregnant in the next 25 months; Lactating women who are breast-feeding; Potential participant lives in the same household as a current participant in this study; Inability to provide blood samples, including difficulty with venous access; Any reason, in the opinion of the Principal Investigator, the participant should not participate.