Stakeholders' Views on Research Design: The Duchenne Muscular Dystrophy Community

利益相关者对研究设计的看法：杜氏肌营养不良症社区

• 批准号: 7527116
• 负责人: ROBERT M NELSON
• 金额: $ 17.99万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-08-01 至 2010-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7527116
• 关键词: Address; Adolescent; Affect; Age; Animals; Attention; Attitude; Back; Benefits and Risks; Canis familiaris; Cardiomyopathies; Cessation of life; Characteristics; Child; Clinical; Clinical Research; Clinical Trials; Collaborations; Communities; Community Participation; Complex; Data; Dependency; Depth; Deterioration; Development; Disease; Disease Progression; Duchenne muscular dystrophy; End Point; Equilibrium; Ethical Issues; Family member; Focus Groups; Gene Transfer; Goals; Grant; Individual; Intervention; Interview; Investigation; Life; Limb structure; Link; Literature; Marketing; Medical; Modeling; Motivation; Muscle; Muscle function; Muscular Dystrophies; Myocardium; Parents; Participant; Patients; Perception; Population; Process; Proteins; Public Health; Publications; Reporting; Research; Research Design; Research Personnel; Respiratory Failure; Risk; Services; Skeletal system; Social Welfare; Staging; Structure; Study Subject; Techniques; Technology; Text; Therapeutic; TimeLine; Toxic effect; Translational Research; Translations; United States National Institutes of Health; Viral Vector; adeno-associated viral vector; advocacy organizations; animal data; boys; community planning; comparative; design; experience; feeding; gene therapy; innovation; member; men; pre-clinical; programs; response; sound; therapy development; trend; vector

DESCRIPTION (provided by applicant): Duchenne Muscular Dystrophy (DMD) is a common X-linked disease in which boys and young men experience progressive skeletal and cardiac muscle deterioration, with loss of ambulation (second decade) and death from cardiomyopathy and respiratory failure (third decade). Animal studies have demonstrated correction of the dystrophy in muscle using gene transfer technology with a viral vector. This technique is now being explored as a means of gene transfer in children with DMD and may substantially increase muscle function and prolong life. However, there are complex ethical issues related to the balance of risk to benefit with the different approaches to gene transfer interventions. This study will explore the views of the DMD community on the risks and benefits of different approaches to gene transfer interventions so that their perspective may be included in ongoing discussions of these ethical issues. The primary goal of this project is to elicit the perspectives of the DMD community (i.e., adolescents with the disease, and their parents) so that this information may be used in designing gene transfer trials for DMD. The specific aims of the study are: (1) To elicit the perspectives of the DMD community on regional versus systemic approaches to gene transfer interventions; (2) To explore whether a commitment to the welfare of the DMD community (i.e., collectivism) influences a hypothetical decision to participate in a study that poses risk and may offer more limited benefit to oneself; and (3) To explore other factors that may influence attitudes towards research participation, such as disease progression, confidence in medical services, level of technological dependency, and individual characteristics such as the mutational cause of the DMD. These aims will be achieved through the use of focus groups with adolescents with DMD, and their parents, followed by individual semi-structured interviews. The proposed research responds directly to two research objectives found in the NIH Action Plan for the Muscular Dystrophies (Dec 2005). The objectives are: (1) evaluate the clinical endpoints needed for and ethical issues associated with gene transfer interventions; and (2) determine the magnitude of changes in endpoints which are clinically meaningful to patients and family members. PUBLIC HEALTH RELEVANCE: A recent report by the NIH Council of Public Representatives recommends that researchers incorporate the "public's perspective [in] the conduct of clinical research and the publication of findings from that research."[8] This study responds to that goal by giving members of the DMD community an opportunity to provide input on the design of gene transfer research intended to benefit those suffering from this devastating disease. The study will provide information to the DMD community about proposed research, gather adolescent and parent views about the risks and benefits of different approaches to gene transfer interventions, and feed this information back to researchers to help them design ethically sound studies that are responsive to community needs.

描述（由申请人提供）：杜氏肌营养不良症（DMD）是一种常见的X连锁疾病，其中男孩和年轻男性经历进行性骨骼和心肌退化，并伴有截肢（第二个十年）和死于心肌病和呼吸衰竭（第三个十年）。动物研究已经证明，使用病毒载体的基因转移技术可以纠正肌肉营养不良。这项技术目前正在探索作为DMD儿童基因转移的一种手段，可能会大大增加肌肉功能并延长寿命。然而，基因转移干预的不同方法存在着与风险与收益平衡相关的复杂伦理问题。本研究将探讨DMD社区对基因转移干预不同方法的风险和益处的看法，以便他们的观点可以包括在正在进行的这些伦理问题的讨论中。该项目的主要目标是引出DMD社区的观点（即，患有这种疾病的青少年及其父母），以便这些信息可以用于设计DMD的基因转移试验。这项研究的具体目的是：（1）引出DMD社区对区域性与系统性基因转移干预方法的看法;（2）探讨对DMD社区福利的承诺（即，集体主义）影响参与研究的假设决定，这会带来风险，并可能为自己提供更有限的利益;（3）探索可能影响研究参与态度的其他因素，如疾病进展，对医疗服务的信心，技术依赖程度，以及个体特征，如DMD的突变原因。这些目标将通过使用与DMD青少年及其父母的焦点小组来实现，然后进行个人半结构化访谈。该研究直接响应了NIH肌营养不良症行动计划（2005年12月）中的两个研究目标。目标是：（1）评估与基因转移干预相关的临床终点和伦理问题;（2）确定对患者和家庭成员有临床意义的终点变化幅度。公共卫生相关性：美国国立卫生研究院公共代表理事会最近的一份报告建议研究人员在进行临床研究和发表研究结果时纳入“公众的观点”。“[8]这项研究响应了这一目标，为DMD社区的成员提供了一个机会，为基因转移研究的设计提供投入，旨在造福那些患有这种毁灭性疾病的人。该研究将向DMD社区提供有关拟议研究的信息，收集青少年和家长对基因转移干预不同方法的风险和益处的看法，并将这些信息反馈给研究人员，以帮助他们设计符合社区需求的道德合理的研究。