MGNet Pilot Studies Program

MGNet 试点研究计划

• 批准号: 10645051
• 负责人: Linda Louise Kusner
• 金额: $ 10.84万
• 依托单位: GEORGE WASHINGTON UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-01 至 2024-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10645051
• 关键词: Address; Agreement; Applications Grants; Awareness; Basic Science; Biometry; Cholinergic Receptors; Clinical Data; Clinical Research; Clinical Trials; Collaborations; Collection; Communities; Coupled; Development; Discipline; Fc Receptor; Foundations; Funding; Goals; Grant; Immunology; Investigation; MUSK gene; Myasthenia Gravis; Outcome Measure; Patient advocacy; Patient-Focused Outcomes; Peer Review; Pilot Projects; Prediction of Response to Therapy; Rare Diseases; Request for Applications; Research; Research Personnel; Research Project Grants; Research Support; Resources; Science; Services; Specimen; Thymoma; Training; biobank; biomarker discovery; biomarker validation; career; clinical phenotype; clinical trial readiness; early onset; improved; innovation; meetings; process improvement; profession allied to medicine; programs; research and development

The primary purpose of the MGNet pilot study program is to promote development of highly innovative early stage investigations focused on discovery of biomarkers predictive of treatment, improve clinical trial readiness through enhancement of patient-centered outcome measures, or process improvement of clinical trials in myasthenia gravis (MG). The MGNet RDCRC will more precisely define the clinical phenotype of MG subtypes (thymoma-associated, acetylcholine receptor antibody-positive of late- and early-onset, muscle-specific kinase-positive, ACHR- MUSK negative) coupled with biospecimen collection. This rich resource will be available for pilot grant proposals with grant recipients benefiting from MGNet core services in clinical research coordinator training, immunology, and biostatistics. Investigators will also actively collaborate with MGNet administration and investigators to assure successful completion of study milestones. MGNet will request applications on an annual basis and only select the most promising proposals that fulfill MGNet goals. The pilot study program benefits greatly from the collaboration of the MG Foundation of America and Conquer MG, which have agreed to fold in their existing pilot grant programs into the MGNet RDCRC.

MGNet试点研究计划的主要目的是促进高度 创新的早期研究集中在发现预测治疗的生物标志物， 通过加强以患者为中心的结局指标，提高临床试验准备程度，或 重症肌无力（MG）临床试验过程的改进。MGNet RDCRC将更多 精确定义MG亚型的临床表型（胸腺瘤相关，乙酰胆碱 晚发型和早发性受体抗体阳性，肌肉特异性激酶阳性，ACHR- MUSK阴性）与生物标本采集相结合。这一丰富的资源将可用于 试点赠款提案，赠款接受者受益于MGNet核心服务， 研究协调员培训、免疫学和生物统计学。调查人员还将积极 与MGNet管理部门和调查人员合作，确保成功完成 研究里程碑。MGNet将每年要求申请，只选择最多的 实现MGNet目标的有前途的提案。试点研究计划受益匪浅， MG美国基金会和征服MG的合作，已同意折叠 他们现有的试点赠款计划纳入MGNet RDCRC。