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ACTIVE NOT RECRUITING
NCT05537220
PHASE3

Oral N-acetylcysteine for Retinitis Pigmentosa

Sponsor: Johns Hopkins University

View on ClinicalTrials.gov

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

Interventions

DRUG

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.

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