Project 3: Treatment of mucostasis and airways obstruction in CF with a novel mucolytic
项目 3:用新型粘液溶解剂治疗 CF 中的粘膜淤积和气道阻塞
基本信息
- 批准号:9356820
- 负责人:
- 金额:$ 27.21万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2017
- 资助国家:美国
- 起止时间:2017-09-07 至 2022-06-30
- 项目状态:已结题
- 来源:
- 关键词:Abnormal ion transportAdverse eventAffectAgar Gel ElectrophoresisAsthmaBiological AssayBiological MarkersBreathingBronchiectasisCessation of lifeChelating AgentsChronicChronic BronchitisChronic Obstructive Airway DiseaseClinicalClinical TrialsCollectionCross-Over StudiesCysteineCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDNADataDehydrationDetergentsDevelopmentDiseaseDisease modelDoseDrug KineticsExhalationFrequenciesGelGoalsHealthHealthcareHydration statusImpairmentIn VitroIon TransportLaboratoriesLeadLungLung diseasesMagnetic Resonance ImagingMeasurementMeasuresModalityMolecularMonitorMorbidity - disease rateMucinsMucociliary ClearanceMucolyticsMucous body substanceObstructionObstructive Lung DiseasesOutcome MeasurePathogenesisPathologicPatientsPharmaceutical PreparationsPhasePlacebo ControlProcessPropertyPulmonary Cystic FibrosisPulmonary Function Test/Forced Expiratory Volume 1Radionuclide ImagingRandomizedReducing AgentsReportingResearchRespiratory physiologySafetySalineScienceSeveritiesSputumSulfhydryl CompoundsSymptomsSyndromeTestingTherapeuticTherapeutic AgentsWestern Blottingairway obstructionbasecystic fibrosis airwaycystic fibrosis patientsdisabilitydisulfide bondextracellularhealthy volunteerhuman diseaseimprovedindexingmortalitynovelnovel therapeuticsprematurepulmonary functionrecombinant human DNasesafety testingsurfactanttargeted agenttherapy development
项目摘要
PROJECT SUMMARY
Mucus obstruction of airways is a significant pathologic feature of CF lung disease. Available therapies are
only partially effective at relieving this obstruction, and additional treatments are needed. Gel-forming mucin
molecules are a major constituent of CF mucus and to a large degree determines its bio-rheological properties.
The development of inhaled agents that target mucin molecules and improve the transportability of airway
mucus is a rational and attractive therapeutic goal. In this Project, we propose to first test the safety of a novel
inhaled mucolytic in CF subjects in a single ascending dose study. We will then evaluate the ability of this
agent to improve mucus clearance and reduce airways obstruction during two weeks of use, in a placebo
controlled cross-over study in CF. Relevant, novel outcome measures will be utilized to evaluate drug effects
on mucus clearance (gamma scintigraphy) and airways obstruction (19F-MRI with 1H-MRI/UTE), in addition to
traditional clinical trial endpoints. The combination of these functional and structural modalities should greatly
enhance our understanding of drug effects on this heterogeneous lung disease.
项目摘要
粘液性气道阻塞是CF肺病的重要病理特征。现有治疗
仅部分有效缓解该阻塞,并且需要额外的治疗。凝胶形成粘蛋白
分子是CF粘液的主要成分,并在很大程度上决定其生物流变学性质。
以粘蛋白分子为靶点改善气道转运的吸入剂的研究进展
粘液是合理的和有吸引力的治疗目标。在这个项目中,我们建议首先测试一本小说的安全性,
在单次剂量递增研究中,CF受试者吸入粘液溶解剂。然后我们将评估这种能力,
在安慰剂中,在两周的使用期间改善粘液清除和减少气道阻塞的药剂
CF的对照交叉研究。将使用相关的、新的结局指标来评价药物作用
对粘液清除(γ射线照相术)和气道阻塞(19 F-MRI和1H-MRI/UTE)的影响,此外,
传统临床试验终点。这些功能和结构模式的结合,
提高我们对这种异质性肺部疾病的药物作用的理解。
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 }}
SCOTT H DONALDSON其他文献
SCOTT H DONALDSON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('SCOTT H DONALDSON', 18)}}的其他基金
MUCUS CLEARANCE DURING ACUTE EXACERBATIONS OF CYSTIC FIBROSIS
囊性纤维化急性发作期间的粘液清除
- 批准号:
7716866 - 财政年份:2008
- 资助金额:
$ 27.21万 - 项目类别:
Mucus Dehydration and Evolution of CF Lung Disease
粘液脱水和 CF 肺病的演变
- 批准号:
7231813 - 财政年份:2006
- 资助金额:
$ 27.21万 - 项目类别:
COMPREHENSIVE ANALYSIS OF MALNUTRITION IN ADULTS WITH CYSTIC FIBROSIS
成人囊性纤维化营养不良的综合分析
- 批准号:
7377447 - 财政年份:2005
- 资助金额:
$ 27.21万 - 项目类别:
EFFICACY OF AMILORIDE AND HYPERTONIC SALINE IN CYSTIC FIBROSIS
阿米洛利和高渗盐水治疗囊性纤维化的疗效
- 批准号:
7200178 - 财政年份:2004
- 资助金额:
$ 27.21万 - 项目类别:
COMPREHENSIVE ANALYSIS OF MALNUTRITION IN ADULTS WITH CYSTIC FIBROSIS
成人囊性纤维化营养不良的综合分析
- 批准号:
7200252 - 财政年份:2004
- 资助金额:
$ 27.21万 - 项目类别:
ORAL SPI-8811 IN PATIENTS WITH CYSTIC FIBROSIS
囊性纤维化患者口服 SPI-8811
- 批准号:
7200288 - 财政年份:2004
- 资助金额:
$ 27.21万 - 项目类别:
Efficacy of Amiloride and Hypertonic Saline in Cystic Fibrosis
阿米洛利和高渗盐水治疗囊性纤维化的疗效
- 批准号:
6980600 - 财政年份:2003
- 资助金额:
$ 27.21万 - 项目类别:
Comprehensive Analysis of Malnutrition in Adults with Cystic Fibrosis
成人囊性纤维化营养不良的综合分析
- 批准号:
6980693 - 财政年份:2003
- 资助金额:
$ 27.21万 - 项目类别:
Oral SPI-8811 in Patients with Cystic Fibrosis
囊性纤维化患者口服 SPI-8811
- 批准号:
6980723 - 财政年份:2003
- 资助金额:
$ 27.21万 - 项目类别:
相似海外基金
Planar culture of gastrointestinal stem cells for screening pharmaceuticals for adverse event risk
胃肠道干细胞平面培养用于筛选药物不良事件风险
- 批准号:
10707830 - 财政年份:2023
- 资助金额:
$ 27.21万 - 项目类别:
Hospital characteristics and Adverse event Rate Measurements (HARM) Evaluated over 21 years.
医院特征和不良事件发生率测量 (HARM) 经过 21 年的评估。
- 批准号:
479728 - 财政年份:2023
- 资助金额:
$ 27.21万 - 项目类别:
Operating Grants
Analysis of ECOG-ACRIN adverse event data to optimize strategies for the longitudinal assessment of tolerability in the context of evolving cancer treatment paradigms (EVOLV)
分析 ECOG-ACRIN 不良事件数据,以优化在不断发展的癌症治疗范式 (EVOLV) 背景下纵向耐受性评估的策略
- 批准号:
10884567 - 财政年份:2023
- 资助金额:
$ 27.21万 - 项目类别:
AE2Vec: Medical concept embedding and time-series analysis for automated adverse event detection
AE2Vec:用于自动不良事件检测的医学概念嵌入和时间序列分析
- 批准号:
10751964 - 财政年份:2023
- 资助金额:
$ 27.21万 - 项目类别:
Understanding the real-world adverse event risks of novel biosimilar drugs
了解新型生物仿制药的现实不良事件风险
- 批准号:
486321 - 财政年份:2022
- 资助金额:
$ 27.21万 - 项目类别:
Studentship Programs
Pediatric Adverse Event Risk Reduction for High Risk Medications in Children and Adolescents: Improving Pediatric Patient Safety in Dental Practices
降低儿童和青少年高风险药物的儿科不良事件风险:提高牙科诊所中儿科患者的安全
- 批准号:
10676786 - 财政年份:2022
- 资助金额:
$ 27.21万 - 项目类别:
Pediatric Adverse Event Risk Reduction for High Risk Medications in Children and Adolescents: Improving Pediatric Patient Safety in Dental Practices
降低儿童和青少年高风险药物的儿科不良事件风险:提高牙科诊所中儿科患者的安全
- 批准号:
10440970 - 财政年份:2022
- 资助金额:
$ 27.21万 - 项目类别:
Improving Adverse Event Reporting on Cooperative Oncology Group Trials
改进肿瘤学合作组试验的不良事件报告
- 批准号:
10642998 - 财政年份:2022
- 资助金额:
$ 27.21万 - 项目类别:
Planar culture of gastrointestinal stem cells for screening pharmaceuticals for adverse event risk
胃肠道干细胞平面培养用于筛选药物不良事件风险
- 批准号:
10482465 - 财政年份:2022
- 资助金额:
$ 27.21万 - 项目类别:
Expanding and Scaling Two-way Texting to Reduce Unnecessary Follow-Up and Improve Adverse Event Identification Among Voluntary Medical Male Circumcision Clients in the Republic of South Africa
扩大和扩大双向短信,以减少南非共和国自愿医疗男性包皮环切术客户中不必要的后续行动并改善不良事件识别
- 批准号:
10191053 - 财政年份:2020
- 资助金额:
$ 27.21万 - 项目类别: