Pilot/Demonstration Clinical Research Projects Program
试点/示范临床研究项目计划
基本信息
- 批准号:8858726
- 负责人:
- 金额:$ 5万
- 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:2014-08-25 至 2019-07-31
- 项目状态:已结题
- 来源:
- 关键词:AcidsAmmoniaBiological AssayBiological MarkersClinicalClinical ResearchClinical TrialsControlled Clinical TrialsDataDevelopmentDiagnosisDiseaseDouble-Blind MethodEarly DiagnosisFamilyFundingGoalsGrantIntestinesInvestigational DrugsLaboratoriesLeadLongitudinal StudiesMethodsMorbidity - disease rateMutationNeonatal ScreeningOutcomePatient Outcomes AssessmentsPatientsPilot ProjectsPlacebo ControlPreventionProductionProtocols documentationRare DiseasesRegistriesResearch PersonnelResearch Project GrantsSigns and SymptomsTestingTherapeutic InterventionUreasecostdata mininginnovationinstrumentnovelnovel therapeutic interventionprogramsresponsetreatment trialurea cycleweb site
项目摘要
The principal aim of the UCDC pilot/demonstration clinical research program is to provide funding for studies of innovative ideas or hypotheses which, if successful, could ultimately lead to improvement in the prevention, diagnosis and/or treatment of UCD. We propose three pilot projects that were selected from 13 projects submitted in response to our internal RFA by UCDC investigators and one from the DMCC. The first project will conduct a small double blind placebo controlled clinical trial of an investigational drug (acetohydroxamic acid) aimed to block urease activity in the intestinal flora, with the goal of reducing intestinal ammonia production to reduce ammonia load in patients with proximal UCD. The second project proposes to develop a NEXTGEN sequencing methods to identify mutations in the three proximal UCD (NAGSD, CPSI D, OTCD) with the long-term goal of using this method as a second-tier test for expanded newborn screening for these disorders. The third project proposes to expand the current contact registry of the UCDC to obtain patient reported outcomes from registrants on urea cycle relevant sign and symptoms using the PROMISE web site and validate data on treatment setting and assess changes in treatments over the ten years of the UCDC program.
UCDC试点/示范临床研究计划的主要目的是为创新思想或假设的研究提供资金,如果成功,最终可能导致UCD的预防,诊断和/或治疗的改善。我们提出了三个试点项目,这些项目是从UCDC调查人员提交给我们内部RFA的13个项目中选出的,其中一个来自DMCC。第一个项目将进行一项小型双盲安慰剂对照临床试验,研究药物(乙酰异羟肟酸)旨在阻断肠道植物群中的尿素酶活性,目的是减少肠道氨的产生,以降低近端UCD患者的氨负荷。第二个项目提出开发NEXTGEN测序方法,以识别三个近端UCD(NAGSD,CPSI D,OTCD)中的突变,长期目标是使用该方法作为扩大新生儿筛查这些疾病的第二层测试。第三个项目建议扩展UCDC的当前联系登记,以使用PROMISE网站从登记者获得尿素循环相关体征和症状的患者报告结果,并验证治疗设置数据,并评估UCDC计划十年来的治疗变化。
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 }}
MARK L. BATSHAW其他文献
MARK L. BATSHAW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('MARK L. BATSHAW', 18)}}的其他基金
Biomarkers of Neurological Injury and Recovery in Urea Cycle Disorders
尿素循环障碍中神经损伤和恢复的生物标志物
- 批准号:
8858723 - 财政年份:2014
- 资助金额:
$ 5万 - 项目类别:
Overall Adminstration of Rare Diseases Clinical Research Consortia (RDCRC)
罕见病临床研究联盟(RDCRC)的总体管理
- 批准号:
8858731 - 财政年份:2014
- 资助金额:
$ 5万 - 项目类别:
Nitric Oxide Supplementation as a Therapeutic Intervention in Argininosuccinate Lyase Deficiency
补充一氧化氮作为精氨基琥珀酸裂解酶缺乏症的治疗干预措施
- 批准号:
8858725 - 财政年份:2014
- 资助金额:
$ 5万 - 项目类别:
Rare Diseases Clinical Research Consorita (RDCRC) for the RDCR Network
RDCR 网络的罕见疾病临床研究联盟 (RDCRC)
- 批准号:
8536435 - 财政年份:2012
- 资助金额:
$ 5万 - 项目类别:
Investigation of Brain Nitrogen Metabolism in Partial Ornithine Trascarbamylase
部分鸟氨酸转氨甲酰酶脑氮代谢的研究
- 批准号:
8325108 - 财政年份:2011
- 资助金额:
$ 5万 - 项目类别:
Rare Diseases Clinical Research Consortia (RDCRC) for the RDCR Network
罕见疾病临床研究联盟 (RDCRC) 的 RDCR 网络
- 批准号:
7932561 - 财政年份:2009
- 资助金额:
$ 5万 - 项目类别:
相似国自然基金
SIRT5/ammonia信号通路介导适应性自噬在急性心肌梗死中的作用及其机制研究
- 批准号:81900312
- 批准年份:2019
- 资助金额:20.0 万元
- 项目类别:青年科学基金项目
相似海外基金
Powering Small Craft with a Novel Ammonia Engine
用新型氨发动机为小型船只提供动力
- 批准号:
10099896 - 财政年份:2024
- 资助金额:
$ 5万 - 项目类别:
Collaborative R&D
STTR Phase I: Microwave-Enhanced Modular Ammonia Synthesis
STTR 第一阶段:微波增强模块化氨合成
- 批准号:
2335104 - 财政年份:2024
- 资助金额:
$ 5万 - 项目类别:
Standard Grant
ENTICE: Enhanced Ammonia Cracking to Improve Engine Combustion and Emissions
ENTICE:增强氨裂解以改善发动机燃烧和排放
- 批准号:
10096979 - 财政年份:2024
- 资助金额:
$ 5万 - 项目类别:
Collaborative R&D
Manufacturing conductive oxides as catalyst support for energy efficient production of hydrogen and ammonia
制造导电氧化物作为氢和氨节能生产的催化剂载体
- 批准号:
2904783 - 财政年份:2024
- 资助金额:
$ 5万 - 项目类别:
Studentship
Development of in situ ammonia capture for enhanced catalytic ammonia synthesis
开发用于增强催化氨合成的原位氨捕获
- 批准号:
24K17765 - 财政年份:2024
- 资助金额:
$ 5万 - 项目类别:
Grant-in-Aid for Early-Career Scientists
EAGER: Enhancement of Ammonia combustion by spatiotemporal control of plasma kinetics
EAGER:通过等离子体动力学的时空控制增强氨燃烧
- 批准号:
2337461 - 财政年份:2024
- 资助金额:
$ 5万 - 项目类别:
Standard Grant
Novel High-Efficiency Ammonia engine Technology for Heavy Duty marine applications (HEAT-HD)
适用于重型船舶应用的新型高效氨发动机技术 (HEAT-HD)
- 批准号:
10096638 - 财政年份:2024
- 资助金额:
$ 5万 - 项目类别:
Collaborative R&D
Three-dimensional solar-energy-driven hydrogen generation from ammonia
三维太阳能驱动的氨制氢
- 批准号:
DP240102787 - 财政年份:2024
- 资助金额:
$ 5万 - 项目类别:
Discovery Projects
Accurate Determination of Branching Fractions in Ammonia Combustion
氨燃烧中支化分数的准确测定
- 批准号:
2329341 - 财政年份:2024
- 资助金额:
$ 5万 - 项目类别:
Standard Grant
Membrane-based real-time ammonia monitoring system for sewage treatment
污水处理膜式氨氮实时监测系统
- 批准号:
IE230100593 - 财政年份:2023
- 资助金额:
$ 5万 - 项目类别:
Early Career Industry Fellowships