NCT07290530

24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome

A 24-Month, Randomized, Double-Masked, Placebo-Controlled Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 80 (estimated)
• Sponsor: Nacuity Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Conditions

• Retinitis Pigmentosa (RP)
• Usher Syndrome

Interventions

Type	Name	Description
DRUG	N-acetylcysteine amide	N-acetylcysteine amide (NPI-001) tablets (250 mg), taken BID
OTHER	Placebo	Placebo tablets, BID

Timeline

• Start date: 2026-06-01
• Primary completion: 2028-12-01
• Completion: 2028-12-01
• First posted: 2025-12-18
• Last updated: 2025-12-18

Regulatory

• FDA-regulated drug study