Safety and Feasibility Study of Transvenous Limb Perfusion with Normal Saline in

生理盐水经肢体静脉灌注的安全性和可行性研究

基本信息

  • 批准号:
    7938901
  • 负责人:
  • 金额:
    $ 22.37万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    2009
  • 资助国家:
    美国
  • 起止时间:
    2009-09-01 至 2013-08-31
  • 项目状态:
    已结题

项目摘要

Safety and Feasibility Study of Transvenous Limb Perfusion with Normal Saline in Human Muscular Dystrophy Due to their genetic basis and the current lack of curative therapy, the muscular dystrophies are excellent candidate diseases for gene therapies. An essential step in this development of such therapies is delivery of genetic material to a single limb to demonstrate safety and efficacy prior to systemic administration. Of the various methods of single limb delivery studied in experimental animals, high-pressure, high-volume transvenous limb perfusion shows the greatest potential to be an effective, clinically practical and generally applicable. In this project, we will perform a safety and feasibility study of transvenous single limb perfusion with normal saline in human subjects with muscular dystrophy. The study is designed as a dose-escalation safety study with the perfusion parameters increased in a stepwise manner and careful monitoring for both bcal and systemic toxicity. This design will also permit us to address the multiple logistical aspects inherent in going from animals to humans with muscular dystrophy including analgesia, vascular access and larger infusion volumes. In Specific Aim 1, we will study young adults with limb-girdle or Becker muscular dystrophy beginning with infusions of .05 mL saline /ml of limb volume and escalating to a maximum of .40 mL/mL. An independent safety monitor will review data on each subject (perfusion parameters, laboratory and clinical testing) and must approve planned perfusion parameters for the next subject. From this study, we will determine the maximum perfusion parameters that are safe and document the degree of fluid delivery into muscle by T2 MRI. In Specific Aim 2, we will carry out a similar dose escalation study in children with Duchenne muscular dystrophy beginning at .05 ml saline /ml of limb volume and escalating to the maximum volume determined from Specific Aim 1 as posing no greater than minimal risk. This study will provide the necessary safety and feasibility data on the perfusion technique itself to provide the basis for future regional limb delivery studies of active gene therapy. The results will be generally applicable to the single limb delivery of many different agents (oligonucleotides, plasmid-DNA and viral delivery systems) and to patients of different ages with diverse types of muscular dystrophy.
生理盐水肢体静脉灌注治疗人类肌营养不良症的安全性和可行性研究 由于它们的遗传基础和目前缺乏治疗方法,肌营养不良症是非常好的。 基因治疗的候选疾病。这种疗法发展的一个重要步骤是提供 在全身给药之前,将遗传物质移植到单个肢体,以证明其安全性和有效性。中的 在实验动物中研究的各种单肢分娩方法,高压,大容量 经静脉肢体灌注显示出最大的潜力,是一种有效的、临床实用的和普遍的 适用。在本项目中,我们将进行经静脉单侧肢体灌注的安全性和可行性研究。 肌肉营养不良的受试者用生理盐水。这项研究被设计为剂量递增 以循序渐进的方式增加灌注参数并对两者进行仔细监测的安全性研究 BCAL和全身毒性。这一设计还将允许我们解决固有的多个后勤方面 从动物到人类的肌营养不良症,包括止痛,血管通路和更大的 输液量。在具体目标1中,我们将研究患有四肢带状或Becker肌营养不良症的年轻人。 从输注0.05毫升生理盐水/毫升肢体体积开始,逐渐增加到最高.40毫升/毫升。一个 独立的安全监测器将审查每个对象(灌注参数、实验室和临床)的数据 测试),并且必须批准下一受试者的计划灌流参数。从这项研究中,我们将 确定安全的最大灌注参数,并记录液体输送到 T2MRI检查肌肉。在具体目标2中,我们将在儿童中进行类似的剂量递增研究 Duchenne肌营养不良始于0.05毫升生理盐水/毫升肢体体积,并升级到 根据具体目标1确定的不大于最小风险的最大交易量。这项研究将 提供有关灌流技术本身的必要的安全性和可行性数据,为 未来主动基因治疗的区域性肢体输送研究。结果将普遍适用于 多种不同制剂(寡核苷酸、质粒DNA和病毒输送系统)的单肢输送 适用于不同年龄、不同类型的肌营养不良症患者。

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 }}

WILLIAM J POWERS其他文献

WILLIAM J POWERS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('WILLIAM J POWERS', 18)}}的其他基金

Training & Education
训练
  • 批准号:
    7938904
  • 财政年份:
    2009
  • 资助金额:
    $ 22.37万
  • 项目类别:
CAROTID OCCLUSION SURGERY STUDY
颈动脉闭塞手术研究
  • 批准号:
    7696299
  • 财政年份:
    2008
  • 资助金额:
    $ 22.37万
  • 项目类别:
Training & Education
训练
  • 批准号:
    7693003
  • 财政年份:
    2008
  • 资助金额:
    $ 22.37万
  • 项目类别:
Safety and Feasibility Study of Transvenous Limb Perfusion with Normal Saline in
生理盐水经肢体静脉灌注的安全性和可行性研究
  • 批准号:
    7535879
  • 财政年份:
    2008
  • 资助金额:
    $ 22.37万
  • 项目类别:
Cerbral Vascular and Metabolic Mechanisms in Alzheimer's Disease
阿尔茨海默病的脑血管和代谢机制
  • 批准号:
    7029787
  • 财政年份:
    2006
  • 资助金额:
    $ 22.37万
  • 项目类别:
Vascular and Metabolic Mechanisms in Alzheimer's Disease
阿尔茨海默病的血管和代谢机制
  • 批准号:
    6901500
  • 财政年份:
    2005
  • 资助金额:
    $ 22.37万
  • 项目类别:
AUTOREGULATION OF CEREBRAL BLOOD FLOW IN ACUTE ISCHEMIA
急性缺血时脑血流的自动调节
  • 批准号:
    6795650
  • 财政年份:
    2003
  • 资助金额:
    $ 22.37万
  • 项目类别:
CEREBRAL MITOCHONDRIAL METABOLISM IN NEURODEGENERATION
神经退行性变中的大脑线粒体代谢
  • 批准号:
    6344240
  • 财政年份:
    2001
  • 资助金额:
    $ 22.37万
  • 项目类别:
CORE--PET FACILITIES
核心--宠物设施
  • 批准号:
    6494886
  • 财政年份:
    2001
  • 资助金额:
    $ 22.37万
  • 项目类别:
CEREBRAL MITOCHONDRIAL METABOLISM IN NEURODEGENERATION
神经退行性变中的大脑线粒体代谢
  • 批准号:
    6540477
  • 财政年份:
    2001
  • 资助金额:
    $ 22.37万
  • 项目类别:

相似海外基金

Rational design of rapidly translatable, highly antigenic and novel recombinant immunogens to address deficiencies of current snakebite treatments
合理设计可快速翻译、高抗原性和新型重组免疫原,以解决当前蛇咬伤治疗的缺陷
  • 批准号:
    MR/S03398X/2
  • 财政年份:
    2024
  • 资助金额:
    $ 22.37万
  • 项目类别:
    Fellowship
Re-thinking drug nanocrystals as highly loaded vectors to address key unmet therapeutic challenges
重新思考药物纳米晶体作为高负载载体以解决关键的未满足的治疗挑战
  • 批准号:
    EP/Y001486/1
  • 财政年份:
    2024
  • 资助金额:
    $ 22.37万
  • 项目类别:
    Research Grant
CAREER: FEAST (Food Ecosystems And circularity for Sustainable Transformation) framework to address Hidden Hunger
职业:FEAST(食品生态系统和可持续转型循环)框架解决隐性饥饿
  • 批准号:
    2338423
  • 财政年份:
    2024
  • 资助金额:
    $ 22.37万
  • 项目类别:
    Continuing Grant
Metrology to address ion suppression in multimodal mass spectrometry imaging with application in oncology
计量学解决多模态质谱成像中的离子抑制问题及其在肿瘤学中的应用
  • 批准号:
    MR/X03657X/1
  • 财政年份:
    2024
  • 资助金额:
    $ 22.37万
  • 项目类别:
    Fellowship
CRII: SHF: A Novel Address Translation Architecture for Virtualized Clouds
CRII:SHF:一种用于虚拟化云的新型地址转换架构
  • 批准号:
    2348066
  • 财政年份:
    2024
  • 资助金额:
    $ 22.37万
  • 项目类别:
    Standard Grant
BIORETS: Convergence Research Experiences for Teachers in Synthetic and Systems Biology to Address Challenges in Food, Health, Energy, and Environment
BIORETS:合成和系统生物学教师的融合研究经验,以应对食品、健康、能源和环境方面的挑战
  • 批准号:
    2341402
  • 财政年份:
    2024
  • 资助金额:
    $ 22.37万
  • 项目类别:
    Standard Grant
The Abundance Project: Enhancing Cultural & Green Inclusion in Social Prescribing in Southwest London to Address Ethnic Inequalities in Mental Health
丰富项目:增强文化
  • 批准号:
    AH/Z505481/1
  • 财政年份:
    2024
  • 资助金额:
    $ 22.37万
  • 项目类别:
    Research Grant
ERAMET - Ecosystem for rapid adoption of modelling and simulation METhods to address regulatory needs in the development of orphan and paediatric medicines
ERAMET - 快速采用建模和模拟方法的生态系统,以满足孤儿药和儿科药物开发中的监管需求
  • 批准号:
    10107647
  • 财政年份:
    2024
  • 资助金额:
    $ 22.37万
  • 项目类别:
    EU-Funded
Ecosystem for rapid adoption of modelling and simulation METhods to address regulatory needs in the development of orphan and paediatric medicines
快速采用建模和模拟方法的生态系统,以满足孤儿药和儿科药物开发中的监管需求
  • 批准号:
    10106221
  • 财政年份:
    2024
  • 资助金额:
    $ 22.37万
  • 项目类别:
    EU-Funded
Recite: Building Research by Communities to Address Inequities through Expression
背诵:社区开展研究,通过表达解决不平等问题
  • 批准号:
    AH/Z505341/1
  • 财政年份:
    2024
  • 资助金额:
    $ 22.37万
  • 项目类别:
    Research Grant
{{ showInfoDetail.title }}

作者:{{ showInfoDetail.author }}

知道了