24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associat… (NCT07290530) | Clinical Trial Compass
Not Yet RecruitingPhase 3
24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
80 participantsStarted 2026-06
Plain-language summary
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 Visit the clinic 9 times for checkups and tests
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Able to comprehend and willing to sign an informed consent form (ICF) and to adhere to the study protocol.
✓. Diagnosed with Usher syndrome.
✓. EZ zone width ≥ 500 microns, which includes the fovea in each eye at Visit 1 (Screening).
✓. 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).
Exclusion criteria
✕. Concurrent retinal pathologies that result in vision loss or inability to fixate, including but not limited to, choroideremia, retinal vein occlusion, and neovascular age-related macular degeneration.
✕. Intraocular surgery within the last two months or capsulotomy within the last month.
✕. 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.
✕. Likely to require cataract surgery within the next 12 months.
✕. Unstable fixation during microperimetry in either eye at either Screening or Baseline visits.
✕. 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.
✕. 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.