The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN)
全球脑白质营养不良倡议临床试验网络 (GLIA-CTN)
基本信息
- 批准号:10266084
- 负责人:
- 金额:$ 140.57万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2019
- 资助国家:美国
- 起止时间:2019-09-30 至 2024-06-30
- 项目状态:已结题
- 来源:
- 关键词:AdrenomyeloneuropathyAdultAdvocacyAdvocateAffectAlexander DiseaseAtaxiaAwarenessAxonBioinformaticsBiological MarkersBirthBloodBrainCaringClassificationClinicalClinical Trials DesignClinical Trials NetworkCollaborationsCollectionCommunitiesCompanionsComputerized Medical RecordCost SharingDataDiagnosisDiseaseDocumentationEarly DiagnosisEarly identificationEarly treatmentEpidemiologistEquilibriumEtiologyFamilyFoundationsFutureGaitGene ExpressionGlial Fibrillary Acidic ProteinHealth ExpendituresHealth PersonnelHealthcareHereditary DiseaseIndividualInstitutionInterferonsJointsKnowledgeLeadershipLongterm Follow-upMeasurementMeasuresMembrane LipidsMetachromatic LeukodystrophyMethodologyMissionModelingMolecularMorbidity - disease rateMyelinNatural HistoryNeonatal ScreeningNervous system structureOutcomePathway interactionsPatient Outcomes AssessmentsPatient advocacyPatientsPelizaeus-Merzbacher DiseasePerformancePhasePilot ProjectsPositioning AttributeProgram DevelopmentProgram SustainabilityPublicationsQuestionnairesRare DiseasesRecording of previous eventsReproducibilityResearchResearch PersonnelResourcesScientistSeverity of illnessSignal TransductionStandardizationSubgroupSurrogate MarkersSyndromeSystemTechnologyTherapeuticTherapeutic TrialsTimeTrainingTranslatingbasecare burdencareer developmentclinical careclinical centerclinical developmentclinical outcome assessmentclinical research siteclinical trial readinessdata integrationdata sharingdisease classificationepidemiology studyimprovedindustry partnerinnovationleukodystrophymeetingsnext generationnext generation sequencingnovelpatient advocacy grouppatient orientedpreclinical developmentprogramsprospectivepseudotoxoplasmosis syndromerate of changesharing platformtoolwearable devicewhite matterworking group
项目摘要
Abstract:
Leukodystrophies are inherited diseases that affect the white matter of the brain due to the loss or absence of
myelin, the lipid membrane that insulates axons in the nervous systems. The leukodystrophy community is at a
key turning point. Novel molecular approaches have increased disease recognition. At the same time key
disorders now have potential treatment pathways. There is urgent unmet need in clinical trial readiness, lest
the promise in pre-clinical development be unable to be translated to well-designed clinical trials. This gap in
knowledge has galvanized stakeholders in the disease community. In collaboration with advocacy partners, we
have formed a research-based consortium, the Global Leukodystrophy InitiAtive Clinical Trials Network
(GLIA-CTN). In this proposal, the GLIA-CTN will embark on a longitudinal natural history, focused on but not
limited to five leukodystrophy with clinical trial readiness: Adrenomyeloneuropathy (AMN), Aicard Goutieres
Syndrome (AGS), Alexander Disease (AxD), Metachromatic Leukodystrophy and Pelizaeus Merzbacher
Disease. Approaches will include Clinical Outcome Assessments (COA) and Patient Reported Outcomes
(PRO) with a focus on reliability, reproducibility and validity. Additionally, we will develop methodology around
Electronic Medical Record (EMR) extraction of natural history data for the leukodystrophies. Companion
projects around novel tools and scales to define the ataxia seen in AMN, novel disease classification strategies
in AGS and their correlation with disease severity and biomarkers, and evidentiary criteria around GFAP (glial
fibrillary acidic protein) as a biomarker for AxD complete the proposal. Oversight for these projects will occur
under a well-developed Administrative Unit which includes a Data Integration Core staffed by bioinformatics,
epidemiologists and biostatisticians thanks to significant cost sharing with participating institutions.
Sustainability of the program is supplemented with a strong career development and pilot project program. All
aspects of the program are carefully aligned with our patient advocacy partners who have provided meaningful
input and will be stakeholders in data sharing platforms. Together, these approaches will establish a platform
to develop clinical trial readiness across the leukodystrophies, in close partnership between clinician scientists,
advocacy groups and industry partners, enabling transformation of the field.
文摘:
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)
数据更新时间:{{ journalArticles.updateTime }}
{{
item.title }}
{{ item.translation_title }}
- DOI:
{{ item.doi }} - 发表时间:
{{ item.publish_year }} - 期刊:
- 影响因子:{{ item.factor }}
- 作者:
{{ item.authors }} - 通讯作者:
{{ item.author }}
数据更新时间:{{ journalArticles.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ monograph.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ sciAawards.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ conferencePapers.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ patent.updateTime }}
FLORIAN S EICHLER其他文献
FLORIAN S EICHLER的其他文献
{{
item.title }}
{{ item.translation_title }}
- DOI:
{{ item.doi }} - 发表时间:
{{ item.publish_year }} - 期刊:
- 影响因子:{{ item.factor }}
- 作者:
{{ item.authors }} - 通讯作者:
{{ item.author }}
{{ truncateString('FLORIAN S EICHLER', 18)}}的其他基金
The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN)
全球脑白质营养不良倡议临床试验网络 (GLIA-CTN)
- 批准号:
10704432 - 财政年份:2019
- 资助金额:
$ 140.57万 - 项目类别:
Myelin Disorders Biorepository Project (MDBP) at the Biospecimen Exchange for Neurological Disorders (BioSEND)
神经疾病生物样本交换中心 (BioSEND) 的髓磷脂疾病生物储存库项目 (MDBP)
- 批准号:
10850332 - 财政年份:2019
- 资助金额:
$ 140.57万 - 项目类别:
Utility of advanced MRI and wearable technology to identify sensory motor outcome measures in leukoencephalopathy with brainstem and spinal cord involvement and lactate elevation (LBSL)
利用先进的 MRI 和可穿戴技术来确定脑干和脊髓受累以及乳酸升高 (LBSL) 的白质脑病的感觉运动结果测量
- 批准号:
10406744 - 财政年份:2019
- 资助金额:
$ 140.57万 - 项目类别:
The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN)
全球脑白质营养不良倡议临床试验网络 (GLIA-CTN)
- 批准号:
9804283 - 财政年份:2019
- 资助金额:
$ 140.57万 - 项目类别:
The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN)
全球脑白质营养不良倡议临床试验网络 (GLIA-CTN)
- 批准号:
10675461 - 财政年份:2019
- 资助金额:
$ 140.57万 - 项目类别:
The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN)
全球脑白质营养不良倡议临床试验网络 (GLIA-CTN)
- 批准号:
10442668 - 财政年份:2019
- 资助金额:
$ 140.57万 - 项目类别:
The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN)
全球脑白质营养不良倡议临床试验网络 (GLIA-CTN)
- 批准号:
10023202 - 财政年份:2019
- 资助金额:
$ 140.57万 - 项目类别:
Safety, Tolerability and Biological Activity of L-serine in HSAN1.
HSAN1 中 L-丝氨酸的安全性、耐受性和生物活性。
- 批准号:
8720080 - 财政年份:2013
- 资助金额:
$ 140.57万 - 项目类别:
相似海外基金
Co-designing a lifestyle, stop-vaping intervention for ex-smoking, adult vapers (CLOVER study)
为戒烟的成年电子烟使用者共同设计生活方式、戒烟干预措施(CLOVER 研究)
- 批准号:
MR/Z503605/1 - 财政年份:2024
- 资助金额:
$ 140.57万 - 项目类别:
Research Grant
Early Life Antecedents Predicting Adult Daily Affective Reactivity to Stress
早期生活经历预测成人对压力的日常情感反应
- 批准号:
2336167 - 财政年份:2024
- 资助金额:
$ 140.57万 - 项目类别:
Standard Grant
RAPID: Affective Mechanisms of Adjustment in Diverse Emerging Adult Student Communities Before, During, and Beyond the COVID-19 Pandemic
RAPID:COVID-19 大流行之前、期间和之后不同新兴成人学生社区的情感调整机制
- 批准号:
2402691 - 财政年份:2024
- 资助金额:
$ 140.57万 - 项目类别:
Standard Grant
Migrant Youth and the Sociolegal Construction of Child and Adult Categories
流动青年与儿童和成人类别的社会法律建构
- 批准号:
2341428 - 财政年份:2024
- 资助金额:
$ 140.57万 - 项目类别:
Standard Grant
Elucidation of Adult Newt Cells Regulating the ZRS enhancer during Limb Regeneration
阐明成体蝾螈细胞在肢体再生过程中调节 ZRS 增强子
- 批准号:
24K12150 - 财政年份:2024
- 资助金额:
$ 140.57万 - 项目类别:
Grant-in-Aid for Scientific Research (C)
Understanding how platelets mediate new neuron formation in the adult brain
了解血小板如何介导成人大脑中新神经元的形成
- 批准号:
DE240100561 - 财政年份:2024
- 资助金额:
$ 140.57万 - 项目类别:
Discovery Early Career Researcher Award
RUI: Evaluation of Neurotrophic-Like properties of Spaetzle-Toll Signaling in the Developing and Adult Cricket CNS
RUI:评估发育中和成年蟋蟀中枢神经系统中 Spaetzle-Toll 信号传导的神经营养样特性
- 批准号:
2230829 - 财政年份:2023
- 资助金额:
$ 140.57万 - 项目类别:
Standard Grant
Usefulness of a question prompt sheet for onco-fertility in adolescent and young adult patients under 25 years old.
问题提示表对于 25 岁以下青少年和年轻成年患者的肿瘤生育力的有用性。
- 批准号:
23K09542 - 财政年份:2023
- 资助金额:
$ 140.57万 - 项目类别:
Grant-in-Aid for Scientific Research (C)
Identification of new specific molecules associated with right ventricular dysfunction in adult patients with congenital heart disease
鉴定与成年先天性心脏病患者右心室功能障碍相关的新特异性分子
- 批准号:
23K07552 - 财政年份:2023
- 资助金额:
$ 140.57万 - 项目类别:
Grant-in-Aid for Scientific Research (C)
Issue identifications and model developments in transitional care for patients with adult congenital heart disease.
成人先天性心脏病患者过渡护理的问题识别和模型开发。
- 批准号:
23K07559 - 财政年份:2023
- 资助金额:
$ 140.57万 - 项目类别:
Grant-in-Aid for Scientific Research (C)