Oral N-acetylcysteine for Retinitis Pigmentosa (NAC Attack)

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ClinicalTrials.gov Identifier: NCT05537220

Recruitment Status : Recruiting

First Posted : September 13, 2022

Last Update Posted : April 12, 2024

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View this study on the modernized ClinicalTrials.gov

Sponsor:

Collaborators:

National Eye Institute (NEI)

Duke University

Emory University

Massachusetts Eye and Ear Infirmary

Mayo Clinic

Medical College of Wisconsin

Retina Foundation of the Southwest

Stanford University

University of California, Davis

University of Florida

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

University of Houston

Medical University of Graz

McGill University

Universität Tübingen

Centro Medico ABC

Radboud University Medical Center

University of Amsterdam

University Hospital, Basel, Switzerland

University College London Hospitals

Northwestern University

University of Pennsylvania

Information provided by (Responsible Party):

Johns Hopkins University

No Study Results Posted on ClinicalTrials.gov for this Study

About Study Results Reporting on ClinicalTrials.gov

Recruitment Status :	Recruiting
Estimated Primary Completion Date :	December 2028
Estimated Study Completion Date :	December 2028

Publications:

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Vithana EN, Abu-Safieh L, Allen MJ, Carey A, Papaioannou M, Chakarova C, Al-Maghtheh M, Ebenezer ND, Willis C, Moore AT, Bird AC, Hunt DM, Bhattacharya SS. A human homolog of yeast pre-mRNA splicing gene, PRP31, underlies autosomal dominant retinitis pigmentosa on chromosome 19q13.4 (RP11). Mol Cell. 2001 Aug;8(2):375-81. doi: 10.1016/s1097-2765(01)00305-7.

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Daiger SP, Sullivan LS, Bowne SJ. Genes and mutations causing retinitis pigmentosa. Clin Genet. 2013 Aug;84(2):132-41. doi: 10.1111/cge.12203. Epub 2013 Jun 19.

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Usui S, Oveson BC, Lee SY, Jo YJ, Yoshida T, Miki A, Miki K, Iwase T, Lu L, Campochiaro PA. NADPH oxidase plays a central role in cone cell death in retinitis pigmentosa. J Neurochem. 2009 Aug;110(3):1028-37. doi: 10.1111/j.1471-4159.2009.06195.x. Epub 2009 May 30.

Shen J, Yang X, Dong A, Petters RM, Peng YW, Wong F, Campochiaro PA. Oxidative damage is a potential cause of cone cell death in retinitis pigmentosa. J Cell Physiol. 2005 Jun;203(3):457-64. doi: 10.1002/jcp.20346.

Komeima K, Usui S, Shen J, Rogers BS, Campochiaro PA. Blockade of neuronal nitric oxide synthase reduces cone cell death in a model of retinitis pigmentosa. Free Radic Biol Med. 2008 Sep 15;45(6):905-12. doi: 10.1016/j.freeradbiomed.2008.06.020. Epub 2008 Jun 28.

Komeima K, Rogers BS, Lu L, Campochiaro PA. Antioxidants reduce cone cell death in a model of retinitis pigmentosa. Proc Natl Acad Sci U S A. 2006 Jul 25;103(30):11300-5. doi: 10.1073/pnas.0604056103. Epub 2006 Jul 18.

Komeima K, Rogers BS, Campochiaro PA. Antioxidants slow photoreceptor cell death in mouse models of retinitis pigmentosa. J Cell Physiol. 2007 Dec;213(3):809-15. doi: 10.1002/jcp.21152.

Lee SY, Usui S, Zafar AB, Oveson BC, Jo YJ, Lu L, Masoudi S, Campochiaro PA. N-Acetylcysteine promotes long-term survival of cones in a model of retinitis pigmentosa. J Cell Physiol. 2011 Jul;226(7):1843-9. doi: 10.1002/jcp.22508.

Ait-Ali N, Fridlich R, Millet-Puel G, Clerin E, Delalande F, Jaillard C, Blond F, Perrocheau L, Reichman S, Byrne LC, Olivier-Bandini A, Bellalou J, Moyse E, Bouillaud F, Nicol X, Dalkara D, van Dorsselaer A, Sahel JA, Leveillard T. Rod-derived cone viability factor promotes cone survival by stimulating aerobic glycolysis. Cell. 2015 May 7;161(4):817-32. doi: 10.1016/j.cell.2015.03.023.

Petit L, Ma S, Cipi J, Cheng SY, Zieger M, Hay N, Punzo C. Aerobic Glycolysis Is Essential for Normal Rod Function and Controls Secondary Cone Death in Retinitis Pigmentosa. Cell Rep. 2018 May 29;23(9):2629-2642. doi: 10.1016/j.celrep.2018.04.111.

Campochiaro PA, Mir TA. The mechanism of cone cell death in Retinitis Pigmentosa. Prog Retin Eye Res. 2018 Jan;62:24-37. doi: 10.1016/j.preteyeres.2017.08.004. Epub 2017 Sep 27.

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Idiopathic Pulmonary Fibrosis Clinical Research Network; Raghu G, Anstrom KJ, King TE Jr, Lasky JA, Martinez FJ. Prednisone, azathioprine, and N-acetylcysteine for pulmonary fibrosis. N Engl J Med. 2012 May 24;366(21):1968-77. doi: 10.1056/NEJMoa1113354. Epub 2012 May 20.

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Campochiaro PA, Iftikhar M, Hafiz G, Akhlaq A, Tsai G, Wehling D, Lu L, Wall GM, Singh MS, Kong X. Oral N-acetylcysteine improves cone function in retinitis pigmentosa patients in phase I trial. J Clin Invest. 2020 Mar 2;130(3):1527-1541. doi: 10.1172/JCI132990.

Usui S, Komeima K, Lee SY, Jo YJ, Ueno S, Rogers BS, Wu Z, Shen J, Lu L, Oveson BC, Rabinovitch PS, Campochiaro PA. Increased expression of catalase and superoxide dismutase 2 reduces cone cell death in retinitis pigmentosa. Mol Ther. 2009 May;17(5):778-86. doi: 10.1038/mt.2009.47. Epub 2009 Mar 17.

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Kong X, Hafiz G, Wehling D, Akhlaq A, Campochiaro PA. Locus-Level Changes in Macular Sensitivity in Patients with Retinitis Pigmentosa Treated with Oral N-acetylcysteine. Am J Ophthalmol. 2021 Jan;221:105-114. doi: 10.1016/j.ajo.2020.08.002. Epub 2020 Aug 11.

Roberts PA, Gaffney EA, Whiteley JP, Luthert PJ, Foss AJE, Byrne HM. Predictive Mathematical Models for the Spread and Treatment of Hyperoxia-induced Photoreceptor Degeneration in Retinitis Pigmentosa. Invest Ophthalmol Vis Sci. 2018 Mar 1;59(3):1238-1249. doi: 10.1167/iovs.17-23177.

Cai CX, Locke KG, Ramachandran R, Birch DG, Hood DC. A comparison of progressive loss of the ellipsoid zone (EZ) band in autosomal dominant and x-linked retinitis pigmentosa. Invest Ophthalmol Vis Sci. 2014 Oct 23;55(11):7417-22. doi: 10.1167/iovs.14-15013.

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