Trials / NCT05537220
Oral N-acetylcysteine for Retinitis Pigmentosa
Underway, not enrolling Phase 3 Drug / neuroprotection
- Sponsor
- Johns Hopkins University
- Treatment
- N-acetylcysteine; Placebo
- Gene
- Not gene-specific
- Conditions
- Retinitis Pigmentosa
- Ages
- 18 Years to 65 Years
- Enrollment
- 485 participants
- Started
- 2023-10-11
- Main results expected
- 2029-05
- Results posted
- Not yet
- Record last updated
- 2026-06-15
What the study is testing
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.
Where it runs
Austria (1)
- Medical University of Graz, Department of Ophthalmology, Graz, Styria
Canada (1)
- McGill University, The Research Institute of the McGill University Health Center, Montreal, Quebec
Germany (1)
- University of Tübingen, Department für Augenheilkunde, Tübingen, Baden-Wurttemberg
Netherlands (2)
- Radboud University, Radboud University Medical Centre, Nijmegen, Gelderland
- University of Amsterdam, Amsterdam Medical Center, Amsterdam, Northern Holland
Switzerland (1)
- Universitätsspital Basel, Eye Clinic, Basel
United Kingdom (1)
- University College London, Moorfields Eye Hospital, London, England
United States (24)
- University of California - Davis, Department of Ophthalmology & Vision Science, Davis, California
- University of Southern California, Keck School of Medicine, Los Angeles, California
- University of California - San Francisco, Department of Ophthalmology, San Francisco, California
- Stanford University, Byers Eye Institute, Stanford, California
- Vitreo Retinal Associates, Gainesville, Florida
- University of Florida - Jacksonville, UF Health Jacksonville, Jacksonville, Florida
- University of Miami, Bascom Palmer Eye Institute, Miami, Florida
- Emory University, Emory Eye Center, Atlanta, Georgia
- University Of Illinois At Chicago, Chicago, Illinois
- Northwestern University, Evanston, Illinois
- University of Iowa, Carver College of Medicine, Iowa City, Iowa
- Wilmer Eye Institute- Johns Hopkins University, Baltimore, Maryland
- Harvard University, Mass. Eye and Ear, Boston, Massachusetts
- University of Michigan, Kellogg Eye Center, Ann Arbor, Michigan
- University of Minnesota, Department of Ophthalmology and Visual Neurosciences, Minneapolis, Minnesota
- Mayo Clinic, Department of Ophthalmology, Rochester, Minnesota
- University of Oklahoma, Dean McGee Eye Institute, Oklahoma City, Oklahoma
- Scheie Eye Institute, Philadelphia, Pennsylvania
- Vanderbilt University, Vanderbilt Eye Institute, Nashville, Tennessee
- Retina Foundation of the Southwest, Dallas, Texas
- University of Utah, Moran Eye Center, Salt Lake City, Utah
- University of Washington, Department of Ophthalmology, Seattle, Washington
- University of Wisconsin - Madison, McPherson Eye Research Institute, Madison, Wisconsin
- Medical College of Wisconsin, The Eye Institute, Milwaukee, Wisconsin