Dominantly Inherited Alzheimer's Network Trials Unit - Adaptive Prevention Trial
显性遗传性阿尔茨海默病网络试验单位 - 适应性预防试验
基本信息
- 批准号:8605439
- 负责人:
- 金额:$ 552.51万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2014
- 资助国家:美国
- 起止时间:2014-08-01 至 2019-12-31
- 项目状态:已结题
- 来源:
- 关键词:AccountingAddressAlzheimer disease preventionAlzheimer&aposs DiseaseAmyloidAmyloid beta-ProteinAmyloidosisAnimal ModelAntibodiesBiochemicalBiological MarkersBoxingBrainCell modelClinicalClinical TrialsClinical Trials DesignCognitiveCollaborationsCross-Sectional StudiesDataData SetDementiaDevelopmentDiseaseDouble-Blind MethodEffectivenessEnrollmentEnsureExcisionFunctional disorderFundingFutureGeneral PopulationGenetic CounselingGenetic screening methodGoalsGrantHome environmentImageImpaired cognitionIndividualInheritedLeadLettersMeasuresMolecularMono-SMonoclonal AntibodiesMutationObservational StudyOnset of illnessParticipantPathway interactionsPatientsPharmaceutical PreparationsPhasePhase III Clinical TrialsPlacebo ControlPlacebosPopulationPositron-Emission TomographyPreventionPrevention approachProcessProductionProtocols documentationPublishingRandomizedRecruitment ActivityRegistriesResearchResearch InfrastructureRiskSafetySiteSumSymptomsTestingTimeTranslationsUnited States National Institutes of HealthVisitarmbasebeta secretasebeta-site APP cleaving enzyme 1brain volumecomputerizeddesigngenetic pedigreeglucose metabolismimprovedinclusion criteriainhibitor/antagonistinterestmeetingsmutation carrierpreventprimary outcomeresearch studyresponsesecondary outcomesuccesstau Proteinstwo-arm study
项目摘要
DESCRIPTION: Autosomal dominant Alzheimer's disease (AD) has informed the field of AD research about the - Further, molecular and biochemical mechanisms that are believed to underlie the athological basis of AD mutations from autosomal dominant AD have provided animal and cellular models that are utilized to develop anti-Aß drugs. Prevention trials of autosomal dominant AD are likely to pioneer approaches for prevention - trials for all forms of AD. This proposal for a phase 3 pivotal cognitive endpoint trial of the leading amyloid- - beta modifying treatment from the ongoing phase 2 Dominantly Inherited Alzheimer's Network - Trial Unit (DIAN TU) biomarker trial aims to prevent cognitive loss in the autosomal-dominant AD population. Results - will advance the search for surrogate biomarkers and for a pathway to preventing AD in the general population. If this single phase 3 trial is positive, it may lead to te approval of a disease modifying drug for prevention. The DIAN-TU has founded the Collaboration for Alzheimer's Prevention (CAP) with the complementary-Alzheimer Prevention Initiative (API) and the ADCS Anti Amyloid treatment in Asymptomatic AD (A4) to ensure processes are efficient, public dollars are well spent, and results will advance the entire field. The DIAN TU established the DIAN Expanded Registry (www.DIANXR.org) and identified additional potential D-IAN-TU trial sites to enable the larger phase 3 prevention trial by increasing the number of participants to more than 3,000. The first prevention trial, a phase 2 biomarker trial, for people at risk of autosomal dominant AD was launched in December, 2012. The DIAN-TU Adaptive Prevention Trial (APT) leverages the existing infrastructure of the DIAN observational study and the DIAN-TU, and enrolls DIAN-TU participants as well as people at risk of autosomal dominant AD from associated sites. The DIANTU APT is the logical next step of the DIAN observational study. The patients are motivated, as this trial is the first concrete hope most have encountered after decades of research participation. The trial design is a randomized, double-blinded placebo controlled two arm trial of the selected best performing safety, tolerability and biomarker efficacy fibrillar anti-Aß antibody, a soluble anti-Aß antibody,or a beta-secretase inhibitor in 266 (n=133 per arm) asymptomatic autosomal dominant AD mutation carriers. Subjects will receive either drug or placebo for four years to determine clinica and cognitive benefit engagement of the CNS mechanism of action and downstream AD biomarkers. The drug arm will be compared to the placebo arm with the primary outcome of CDR Sum-of-Boxes and secondary outcomes of cognitive measures, computerized cognitive battery, safety, and AD biomarkers.
产品说明:常染色体显性阿尔茨海默病(AD)已经告知AD研究领域关于-此外,被认为是来自常染色体显性AD的AD突变的病理学基础的分子和生化机制已经提供了用于开发抗阿尔茨海默病药物的动物和细胞模型。常染色体显性AD的预防试验可能是预防所有形式AD的先驱方法。这项针对正在进行的2期显性遗传性阿尔茨海默病网络-试验单位(DIAN TU)生物标志物试验的主要淀粉样蛋白-β修饰治疗的3期关键认知终点试验的提案旨在预防常染色体显性AD人群的认知丧失。结果-将推进寻找替代生物标志物和在一般人群中预防AD的途径。如果这一单一的3期试验是积极的,它可能导致批准一种疾病修饰药物用于预防。DIAN-TU与补充性阿尔茨海默病预防倡议(API)和ADCS抗淀粉样蛋白治疗无症状AD(A4)建立了阿尔茨海默病预防合作(CAP),以确保流程有效,公共资金得到合理使用,结果将推动整个领域的发展。DIAN TU建立了DIAN扩展登记研究(www.DIANXR.org),并确定了其他潜在的D-IAN-TU试验中心,通过将受试者人数增加到3,000多人,实现更大规模的III期预防试验。2012年12月启动了针对常染色体显性AD风险人群的第一项预防试验,即2期生物标志物试验。DIAN-TU适应性预防试验(APT)利用DIAN观察性研究和DIAN-TU的现有基础设施,招募DIAN-TU参与者以及相关研究中心的常染色体显性AD风险人群。DIANTU APT是DIAN观察性研究合乎逻辑的下一步。患者们很有动力,因为这项试验是大多数人在参与研究数十年后遇到的第一个具体希望。试验设计是在266名(每组n=133名)无症状常染色体显性AD突变携带者中进行的所选表现最佳的安全性、耐受性和生物标志物功效纤维状抗-A β抗体、可溶性抗-A β抗体或β-分泌酶抑制剂的随机化、双盲安慰剂对照两组试验。受试者将接受药物或安慰剂治疗4年,以确定CNS作用机制和下游AD生物标志物的临床和认知获益。将药物组与安慰剂组进行比较,主要结局为CDR汇总表,次要结局为认知指标、计算机认知成套测验、安全性和AD生物标志物。
项目成果
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{{ truncateString('RANDALL J BATEMAN', 18)}}的其他基金
DIAN-TU: Tau Next Generation Prevention Trial - Administrative Supplement
DIAN-TU:Tau 下一代预防试验 - 行政补充
- 批准号:
10307004 - 财政年份:2020
- 资助金额:
$ 552.51万 - 项目类别:
DIAN-TU: Tau Next Generation Prevention Trial
DIAN-TU:Tau 下一代预防试验
- 批准号:
10261442 - 财政年份:2020
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$ 552.51万 - 项目类别:
DIAN-TU: Tau Next Generation Prevention Trial
DIAN-TU:Tau 下一代预防试验
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10452692 - 财政年份:2020
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Characterization of Neurofilament Light Chain in Alzheimer's Disease and Other Neurodegenerative Disorders
阿尔茨海默病和其他神经退行性疾病中神经丝轻链的表征
- 批准号:
9975558 - 财政年份:2020
- 资助金额:
$ 552.51万 - 项目类别:
Blood amyloid-beta relationship with amyloid plaques and CSF amyloid-beta
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- 批准号:
10077729 - 财政年份:2020
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$ 552.51万 - 项目类别:
DIAN-TU: Tau Next Generation Prevention Trial
DIAN-TU:Tau 下一代预防试验
- 批准号:
10035004 - 财政年份:2020
- 资助金额:
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