Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe)
ALS 及相关疾病的临床研究以促进治疗开发 (CReATe)
基本信息
- 批准号:10020808
- 负责人:
- 金额:$ 163.39万
- 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:2014-09-30 至 2024-08-31
- 项目状态:已结题
- 来源:
- 关键词:AdvocacyAmyotrophic Lateral SclerosisAwardBehavioralBiochemicalBiological MarkersBiological Specimen BanksBiotechnologyCaregiversClinicalClinical DataClinical InvestigatorClinical ResearchClinical TrialsCognitiveCollaborationsCommunitiesCommunity OutreachDataData CollectionDevelopmentDiagnosisDiagnosticDiseaseElectronic Health RecordEnsureEpidemiologyEtiologyEuropeanExtramural ActivitiesFamily memberFosteringFoundationsFrontotemporal DementiaFundingFutureGeneticGoalsGrantHealthHereditary Spastic ParaplegiaHeterogeneityInfrastructureLightMotorNational Institute of Neurological Disorders and StrokeNeurodegenerative DisordersNeurologistNeuronsOutcome MeasurePatient Outcomes AssessmentsPatient ParticipationPatient Participation RatesPatientsPhasePhenotypePhysiciansPrimary Lateral SclerosisProcessProgressive Muscular AtrophyRare DiseasesReadinessRegistriesResearchResearch PersonnelResistanceResourcesRho-associated kinaseSafetySaint Jude Children&aposs Research HospitalScienceScientistSeriesSerumStructureSymptomsSystemTrainingUnited States National Institutes of Healthbasebiobankbiological heterogeneitybiomarker discoverybiomarker validationcandidate markercareercareer developmentcohortdata toolsdisease heterogeneitydrug developmenteffective therapyepigenetic markerfasudilimprovedindexingindustry partnerkinase inhibitormeetingsmultidisciplinarymultisystem proteinopathyneurofilamentneuromuscularoutreachpatient advocacy grouppatient engagementphenotypic dataprogramsresearch and developmentresearch studyskillssuccesstherapeutic developmenttherapy developmenttranslational scientisturinarywhole genome
项目摘要
Project Summary / Abstract
The Clinical Research in ALS and related disorders for Therapy Development (CReATe) Consortium is a Rare
Diseases Clinical Research Consortium (RDCRC) that forms part of the National Institutes of Health Rare
Diseases Clinical Research Network (RDCRN). CReATe comprises a multi-disciplinary group of clinicians,
scientists, educators, patient advocacy groups and other strategic partners, and aims to advance therapeutic
development for patients with amyotrophic lateral sclerosis (ALS) and related disorders including progressive
muscular atrophy (PMA), primary lateral sclerosis (PLS), frontotemporal dementia (FTD), multisystem
proteinopathy (MSP), and hereditary spastic paraplegia (HSP). We recognize that the obstacles to therapeutic
development are multifactorial and include: (a) etiological and biological heterogeneity; (b) phenotypic
heterogeneity; (c) limitations of existing clinical outcome measures for use in early-to-mid phase clinical trials;
(d) a paucity of biomarkers that have been validated as “fit for purpose”); (e) the relatively late stage at which
symptoms appear, diagnosis is made, and treatment is initiated; and (f) variable (often low) rates of patient
participation in clinical research studies. CReATe investigators are engaged in a range of clinical trial
readiness activities that aim to overcome these obstacles, and to thereby advance therapeutic development for
this group of rare diseases. In addition, CReATe aims to promote and support collaborative research in the
field of ALS and related disorders; develop and disseminate resources such as the CReATe Biorepository and
our electronic health record-based ALS Toolkit that are of value to the broader scientific community; lower
barriers to patient participation in research; cultivate and enhance the careers of young clinical investigators
and translational scientists dedicated to the study of this group of rare diseases; and engage both lay- and
scientific-community stakeholders in a partnership that fosters efforts to develop treatments for patients
afflicted with these rare diseases.
项目总结/摘要
ALS和相关疾病治疗开发(CReATe)联盟的临床研究是罕见的
疾病临床研究联盟(RDCRC)是美国国立卫生研究院的一部分,
疾病临床研究网络(RDCRN)。CReATe由多学科的临床医生组成,
科学家,教育工作者,患者倡导团体和其他战略合作伙伴,旨在推进治疗
肌萎缩侧索硬化症(ALS)和相关疾病(包括进行性
肌萎缩(PMA),原发性侧索硬化(PLS),额颞叶痴呆(FTD),多系统
蛋白质病(MSP)和遗传性痉挛性截瘫(HSP)。我们认识到治疗的障碍
发展是多因素的,包括:(a)病因学和生物学异质性;(B)表型
异质性;(c)用于早期至中期临床试验的现有临床结果测量的局限性;
(d)缺乏经验证“符合目的”的生物标志物);(e)
出现症状,作出诊断,并开始治疗;(f)可变(往往很低)的病人率
参与临床研究。CReATe研究者参与了一系列临床试验
准备活动,旨在克服这些障碍,从而推动治疗发展,
这组罕见疾病。此外,CReATe旨在促进和支持在
ALS和相关疾病领域;开发和传播资源,如CReATe生物储存库,
我们基于电子健康记录的ALS工具包对更广泛的科学界有价值;
患者参与研究的障碍;培养和提高年轻临床研究者的职业生涯
和致力于研究这组罕见疾病的转化科学家;并让外行和
科学界利益相关者结成伙伴关系,促进为患者开发治疗方法的努力
患有这些罕见的疾病。
项目成果
期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
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{{ truncateString('Michael Benatar', 18)}}的其他基金
Multi-Center ALS Biomarker Validation Study (CReATe Biomarkers)
多中心 ALS 生物标志物验证研究 (CReATe Biomarkers)
- 批准号:
10410344 - 财政年份:2018
- 资助金额:
$ 163.39万 - 项目类别:
University of Miami NeuroNEXT Trial Site
迈阿密大学 NeuroNEXT 试验网站
- 批准号:
10201771 - 财政年份:2018
- 资助金额:
$ 163.39万 - 项目类别:
Multi-Center ALS Biomarker Validation Study (CReATe Biomarkers)
多中心 ALS 生物标志物验证研究 (CReATe Biomarkers)
- 批准号:
10612967 - 财政年份:2018
- 资助金额:
$ 163.39万 - 项目类别:
University of Miami NeuroNEXT Trial Site
迈阿密大学 NeuroNEXT 试验网站
- 批准号:
10593638 - 财政年份:2018
- 资助金额:
$ 163.39万 - 项目类别:
University of Miami NeuroNEXT Trial Site
迈阿密大学 NeuroNEXT 试验网站
- 批准号:
9980517 - 财政年份:2018
- 资助金额:
$ 163.39万 - 项目类别:
Clinical Centers for the NINDS NeuroNEXT(Network of Excellence in Neuroscience Clinical Trials) Consortium
NINDS NeuroNEXT(神经科学临床试验卓越网络)联盟的临床中心
- 批准号:
10744345 - 财政年份:2018
- 资助金额:
$ 163.39万 - 项目类别:
Pre-Symptomatic Familial ALS (Pre-fALS) Study - Prelude to a Treatment Trial
症状前家族性 ALS (Pre-fALS) 研究 - 治疗试验的前奏
- 批准号:
10237152 - 财政年份:2018
- 资助金额:
$ 163.39万 - 项目类别:
Pre-Symptomatic Familial ALS (Pre-fALS) Study - Prelude to a Treatment Trial
症状前家族性 ALS (Pre-fALS) 研究 - 治疗试验的前奏
- 批准号:
10469619 - 财政年份:2018
- 资助金额:
$ 163.39万 - 项目类别:
Pre-Symptomatic Familial ALS (Pre-fALS) Study - Prelude to a Treatment Trial
症状前家族性 ALS (Pre-fALS) 研究 - 治疗试验的前奏
- 批准号:
10001608 - 财政年份:2018
- 资助金额:
$ 163.39万 - 项目类别:
Multi-Center ALS Biomarker Validation Study (CReATe Biomarkers)
多中心 ALS 生物标志物验证研究 (CReATe Biomarkers)
- 批准号:
9923007 - 财政年份:2018
- 资助金额:
$ 163.39万 - 项目类别:
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