drugset / Trial / NCT05537220
Oral N-acetylcysteine for Retinitis Pigmentosa
Phase 3
Active not recruiting
485 enrolled
Johns Hopkins University
Duke University · collabEmory University · collabMassachusetts Eye and Ear Infirmary · collabMayo Clinic · collabMcGill University · collabMedical College of Wisconsin · collabMedical University of Graz · collabNational Eye Institute (NEI) · collabNorthwestern University · collabRadboud University Medical Center · collabRetina Foundation of the Southwest · collabStanford University · collabUniversity College London Hospitals · collabUniversity Hospital, Basel, Switzerland · collabUniversity of Amsterdam · collabUniversity of California, Davis · collabUniversity of Florida · collabUniversity of Houston · collabUniversity of Iowa · collabUniversity of Miami · collabUniversity of Michigan · collabUniversity of Minnesota · collabUniversity of Oklahoma · collabUniversity of Pennsylvania · collabUniversity of Southern California · collabUniversity of Utah · collabUniversity of Washington · collabUniversity of Wisconsin, Madison · collabUniversität Tübingen · collabVanderbilt University · collabVitreo Retinal Associates, PA · collab
RandomizedParallel-groupQuadruple-blindTreatment
Summary
Retinitis pigmentosa (RP) is an inherited retinal degeneration caused by one of several mistakes in the genetic code. Such mistakes are called mutations. The mutations cause degeneration of rod photoreceptors which are responsible for vision in dim illumination resulting in night blindness. After rod photoreceptors are eliminated, gradual degeneration of cone photoreceptors occurs resulting in gradual constriction of side vision that eventually causes tunnel vision. Oxidative stress contributes to cone degeneration. N-acetylcysteine (NAC) reduces oxidative stress and in animal models of RP it slowed cone degeneration. In a phase I clinical trial in patients with RP, NAC taken by month for 6 months caused some small improvements in two different vision tests suggesting that long-term administration of NAC might slow cone degeneration in RP. NAC Attack is a clinical trial being conducted at many institutions in the US, Canada, and Europe designed to determine if taking NAC for several years provides benefit in patients with RP.
Timeline
- Start
- 2023-10-11
- Primary completion
- 2029-05
- Completion
- 2030-05
Publications
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- Kong X, Ibukun F, Khan MJ, Hafiz G, Wehling D, Dreger K, Singh M, Birch D, Jaffe G, Naufal F, Traboulsi EI, Duncan J, Hufnagel R, Carroll J, Lu Y, Matsui R, Moulton LH, Hawkins B, Campochiaro PA; NAC Attack Study Investigative Group. Protocol for NAC Attack, a phase-3, multicenter randomized, parallel, double masked, placebo controlled trial evaluating the efficacy and safety of oral N-acetylcysteine (NAC) in patients with retinitis pigmentosa. medRxiv [Preprint]. 2025 Nov 6:2025.11.05.25339486. doi: 10.1101/2025.11.05.25339486.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | N-acetylcysteine | Small molecule | 1800 mg | Oral |