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, Mexico, and Europe designed to
determine if taking NAC for several years provides benefit in patients with RP.
Phase:
Phase 3
Details
Lead Sponsor:
Johns Hopkins University
Collaborators:
Centro Medico ABC Duke University Emory University Massachusetts Eye and Ear Infirmary Mayo Clinic McGill University Medical College of Wisconsin Medical University of Graz National Eye Institute (NEI) Northwestern University Radboud University Medical Center Retina Foundation of the Southwest Stanford University Universität Tübingen University College London Hospitals University Hospital, Basel, Switzerland University of Amsterdam University of California, Davis University of Florida University of Houston University of Illinois at Chicago University of Iowa University of Miami University of Michigan University of Minnesota University of Oklahoma University of Southern California University of Utah University of Washington University of Wisconsin, Madison Vanderbilt University Vitreo Retinal Associates, PA