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

NAC Attack，一项针对色素性视网膜炎患者的 3 期、多中心、随机、安慰剂对照试验

• 批准号: 10593911
• 负责人: Xiangrong Kong
• 金额: $ 521.86万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-03-17 至 2027-07-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10593911
• 关键词: Acetylcysteine; Adverse event; Age; Appointments and Schedules; Area; Blindness; Cause of Death; Cell Death; Certification; Clinical; Clinical Management; Clinical Trials Design; Collaborations; Cone; Consumption; Controlled Clinical Trials; DNA Sequence Alteration; Data; Data Collection; Data Management Resources; Databases; Development; Diabetic Retinopathy; Early treatment; Ensure; Eye; Genotype; Human; Human Resources; Image; Incidence; Knowledge; Lead; Leadership; Longitudinal Studies; Maintenance; Masks; Measures; Methods; Molecular; Monitor; Multi-Institutional Clinical Trial; Mutation; Online Systems; Optical Coherence Tomography; Oral; Outcome; Outcome Measure; Oxidative Stress; Participant; Pathogenicity; Patient Outcomes Assessments; Patients; Performance; Perimetry; Persons; Phase; Placebo Control; Placebos; Preparation; Protocols documentation; Randomized; Recording of previous events; Reporting; Research Personnel; Resolution; Retinal Cone; Retinitis Pigmentosa; Role; Safety; Scanning; Secure; Severities; Site Visit; Smoking Status; Statistical Data Interpretation; Subgroup; Symptoms; Testing; Time; Treatment Efficacy; Vision; Visit; Visual; Visual Acuity; Visual Fields; Width; adaptive optics scanning laser ophthalmoscopy; aged; arm; causal variant; clinical research site; cloud based; comparison control; constriction; data management; design; disability; efficacy evaluation; experience; gene therapy; improved; inherited retinal degeneration; loss of function; macula; meetings; member; new technology; oral supplementation; oxidative damage; participant retention; pharmacologic; photoreceptor degeneration; prevent; programs; quality assurance; randomized placebo controlled trial; randomized trial; recruit; retinal rods; sex; standard care; success; targeted treatment; treatment arm; trial design

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.

摘要：色素性视网膜炎（RP）是一种由一种或多种视网膜变性引起的遗传性视网膜变性