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Oral N-acetylcysteine for Retinitis Pigmentosa
Sponsor: Johns Hopkins University
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.
Official title: NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients With Retinitis Pigmentosa
Key Details
Gender
All
Age Range
18 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
485
Start Date
2023-10-11
Completion Date
2030-05
Last Updated
2026-02-27
Healthy Volunteers
No
Conditions
Interventions
N-acetylcysteine
After randomization, participants will be given about 10-months supply of study drug (intervention), with instructions to take 3 effervescent tablets in water twice a day. They will return to the clinic at M4.5 for evaluation and then at M9, M18, M27, M36, M40.5 and M45. At each in-clinic visit, drug reconciliation will occur. At each visit at Baseline, M9, M18, M27, M36, that is, every 9 months, participants will be given another 10-month supply of study drug.
Placebo
After randomization, participants will be given about 10-months supply of placebo, with instructions to take 3 effervescent tablets in water twice a day. They will return to the clinic at M4.5 for evaluation and then at M9, M18, M27, M36, M40.5 and M45. At each in-clinic visit, efficacy and safety assessments will be done and drug reconciliation will occur. At baseline, M9, M18, M27, and M36 participants will be given another 10-month supply of placebo.
Locations (31)
University of California - Davis, Department of Ophthalmology & Vision Science
Davis, California, United States
University of Southern California, Keck School of Medicine
Los Angeles, California, United States
University of California - San Francisco, Department of Ophthalmology
San Francisco, California, United States
Stanford University, Byers Eye Institute
Stanford, California, United States
Vitreo Retinal Associates
Gainesville, Florida, United States
University of Florida - Jacksonville, UF Health Jacksonville
Jacksonville, Florida, United States
University of Miami, Bascom Palmer Eye Institute
Miami, Florida, United States
Emory University, Emory Eye Center
Atlanta, Georgia, United States
University Of Illinois At Chicago
Chicago, Illinois, United States
Northwestern University
Evanston, Illinois, United States
University of Iowa, Carver College of Medicine
Iowa City, Iowa, United States
Wilmer Eye Institute- Johns Hopkins University
Baltimore, Maryland, United States
Harvard University, Mass. Eye and Ear
Boston, Massachusetts, United States
University of Michigan, Kellogg Eye Center
Ann Arbor, Michigan, United States
University of Minnesota, Department of Ophthalmology and Visual Neurosciences
Minneapolis, Minnesota, United States
Mayo Clinic, Department of Ophthalmology
Rochester, Minnesota, United States
University of Oklahoma, Dean McGee Eye Institute
Oklahoma City, Oklahoma, United States
Scheie Eye Institute
Philadelphia, Pennsylvania, United States
Vanderbilt University, Vanderbilt Eye Institute
Nashville, Tennessee, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
University of Utah, Moran Eye Center
Salt Lake City, Utah, United States
University of Washington, Department of Ophthalmology
Seattle, Washington, United States
University of Wisconsin - Madison, McPherson Eye Research Institute
Madison, Wisconsin, United States
Medical College of Wisconsin, The Eye Institute
Milwaukee, Wisconsin, United States
Medical University of Graz, Department of Ophthalmology
Graz, Styria, Austria
McGill University, The Research Institute of the McGill University Health Center
Montreal, Quebec, Canada
University of Tübingen, Department für Augenheilkunde
Tübingen, Baden-Wurttemberg, Germany
Radboud University, Radboud University Medical Centre
Nijmegen, Gelderland, Netherlands
University of Amsterdam, Amsterdam Medical Center
Amsterdam, Northern Holland, Netherlands
Universitätsspital Basel, Eye Clinic
Basel, Switzerland
University College London, Moorfields Eye Hospital
London, England, United Kingdom