Developing Endpoints to Facilitate Clinical Trials in Rare Diseases

开发终点以促进罕见疾病的临床试验

• 批准号: 9052881
• 负责人: Scott R Plotkin
• 金额: $ 1万
• 依托单位: MASSACHUSETTS GENERAL HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-12-04 至 2016-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9052881
• 关键词: Adopted; Area; Benign; Biological Markers; Cancer Therapy Evaluation Program; Child; Child Support; Clinical; Clinical Trials; Cognitive; Collaborations; Communities; Consensus; Country; Discipline; Doctor of Philosophy; Ensure; Evaluation; Fostering; Foundations; Funding; General Hospitals; Goals; Histologic; Image; Individual; International; Lead; Leadership; Malignant Neoplasms; Maryland; Massachusetts; Morbidity - disease rate; National Cancer Institute; Natural History; Nerve Sheath Tumors; Neurocognitive; Neurofibromatoses; Neurofibromatosis 1; Neurofibromatosis 2; Neurology; Newsletter; Outcome; Outcome Measure; Outcomes Research; Patient Outcomes Assessments; Patient-Focused Outcomes; Patients; Peer Review; Pharmacologic Substance; Phase; Play; Predisposition; Publications; Publishing; Rare Diseases; Recommendation; Research; Research Personnel; Review Literature; SDZ RAD; Schwannomatosis; Solid; Specialist; Surrogate Markers; Syndrome; Time; Tuberous Sclerosis; Tumor Suppressor Proteins; Uncertainty; United States; United States Food and Drug Administration; Visual; Work; antitumor agent; design; experience; functional outcomes; interest; meetings; neurogenetics; novel; oncology; posters; public health relevance; response; symposium; trial design; tumor; web site; working group

 DESCRIPTION (provided by applicant): Neurofibromatosis 1 (NF1), neurofibromatosis 2 (NF2), and schwannomatosis are a group of related neurogenetic tumor suppressor syndromes that share a predisposition to develop nerve sheath tumors. Because most tumors encountered in patients with NF1, NF2, and schwannomatosis are histologically benign, overall survival is not an appropriate clinical trial outcome for these patients. In 2011, a group of NF researchers agreed to meet periodically to develop novel outcomes for these conditions. The Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration consists of specialists from multiple countries and disciplines dedicated to the study of neurofibromatosis, schwannomatosis, and clinical trials. REiNS holds an annual conference during the winter in which recommendations for clinical trial outcomes are discussed. Each year, the Leadership Group of REiNS selects experts on the topics under discussion to present the recommendations of each working group for discussion by the larger group. These meetings have led to multiple publications that have been incorporated into ongoing clinical trials and that have been published in the peer-reviewed literature. The annual REiNS meeting is a critical forum for generating new ideas and approaches to these rare tumor suppressor syndromes. The 2015 conference topic is Developing Endpoints to Facilitate Clinical Trials in Rare Diseases and will include outside speakers from the Food and Drug Administration, the Cancer Therapy Evaluation Program, the Children's Tumor Foundation, and the National Cancer Institute.

 描述（由申请人提供）：神经纤维瘤病 1 (NF1)、神经纤维瘤病 2 (NF2) 和神经鞘瘤病是一组相关的神经源性肿瘤抑制综合征，它们具有发生神经鞘瘤的倾向，因为大多数肿瘤发生在 NF1、NF2 患者中。 ，和神经鞘瘤病在组织学上是良性的，总体生存率对于这些患者来说不是一个合适的临床试验结果。 2011年，神经纤维瘤病和神经鞘瘤病反应评估 (REiNS) 国际合作小组由来自多个国家和学科的专家组成，致力于神经纤维瘤病、神经鞘瘤病和临床试验的研究。 REiNS 在冬季举行年度会议，讨论临床试验结果的建议。每年，REiNS 领导小组都会就所讨论的主题选出专家，提出各工作组的建议。这些会议产生了多项出版物，这些出版物已被纳入正在进行的临床试验中。 年度 REiNS 会议是针对这些罕见肿瘤抑制综合征产生新想法和方法的重要论坛，2015 年会议的主题是“开发端点以促进罕见疾病的临床试验”，并将包括外部发言人。来自美国食品和药物管理局、癌症治疗评估计划、儿童肿瘤基金会和国家癌症研究所。