NAC Attack, a phase-3, multicenter, randomized, placebo-controlled trial in patients with retinitis pigmentosa

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ABSTRACT: Retinitis Pigmentosa (RP) is an inherited retinal degeneration caused by one or more of a large number of genetic mutations. It is a major cause of blindness and severe vision loss in people aged 20-60 years. The first symptom in RP is loss of night vision as a result of mutation-caused death of rod photoreceptors. Subsequent loss of cone photoreceptors leads to gradual constriction of visual field and eventually blindness. Currently, there is no treatment for preventing vision loss in RP. NAC Attack is a phase-3 multicenter, randomized, double masked, parallel and placebo-controlled clinical trial to evaluate the efficacy and safety of oral N-acetylcysteine (NAC) in patients with RP. Participants will be randomized to receive either oral NAC of 1800mg/bid or placebo in a ratio of 2:1 and will be followed for 45-months. The Coordinating Center (CC) collaborates closely with the Executive Committee to ensure the success of NAC Attack. The CC contributes to study leadership and provides expertise on trial design, facilitation of recruitment and retention of participants, coordination across all trial entities, implementation and maintenance of a high-quality data management system, statistical analysis, and quality assurance. The CC will:  Enlist and retain Clinical Sites with experienced investigators and sufficient recruitment capacity;  Ensure that Clinical Site personnel complete certification requirements and are in regulatory compliance;  Create and maintain the study database through design and implementation of data collection forms, secure web-based data capture using REDCap, data editing, and data management;  Create and maintain the study cloud-based portal using JHU OneDrive to receive and manage imaging and test files from the clinical sites;  Advise Clinical Sites on resolution of real-time problems and overall strategies for successful implementation of the study protocol;  Provide aids to clinical sites for study management such as appointment schedule, reminders of upcoming visits, missing forms, and incomplete submission of study materials;  Provide regular reports on study progress & performance to Clinical Sites and all study committees;  Design and implement a full program of quality assurance activities including certification of personnel and clinical sites, site visits, and performance monitoring;  Provide interim and final statistical analyses of study data;  Prepare for various committee and study meetings;  Participate and lead in the preparation of scientific presentations and reports. NAC Attack has the potential to identify a pharmacological therapy benefitting all patients with RP, irrespective of the identification of their causative mutation, and thus to impact on the clinical management of RP.