PROSPECTIVE STUDY OF NITRIC OXIDE FOR SICKLE CELL PAIN CRISIS

一氧化氮治疗镰状细胞疼痛危机的前瞻性研究

• 批准号: 7605123
• 负责人: RACHELLE F NUSS
• 金额: $ 1.49万
• 依托单位: UNIVERSITY OF COLORADO DENVER
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-03-01 至 2008-02-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7605123
• 关键词: Agreement; Air; Blinded; Breathing; Cannulas; Clinical; Computer Retrieval of Information on Scientific Projects Database; Double-Blind Method; End Point; Enrollment; Funding; Gases; Goals; Grant; Hospitals; Hour; Incidence; Industry; Institution; Nitric Oxide; Nitrogen; Nose; Oxygen; Pain; Patients; Persons; Physiologic pulse; Placebos; Population Study; Prospective Studies; Pulse taking; Rank-Sum Tests; Reporting; Research; Research Personnel; Resolution; Resources; Safety; Sickle Cell; Sickle Cell Anemia; Source; System; Testing; Therapeutic; Time; United States National Institutes of Health; beta Thalassemia; face mask; inhaled nitric oxide; prospective; randomized placebo controlled trial; research and development

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The study is an investigator-initiated and industry/intramural NIH sponsored trial involving a collaborative research and development agreement between NIH and INO Therapeutics. The goal is to determine the safety and efficacy of nitric oxide (NO) for inhalation in the treatment of vaso-occlusive pain crisis (VOC) in patients with sickle cell disease. It is a prospective, multicenter, double-blind, randomized, placebo-controlled trial sponsored by INO Therapeutics with the study initiated by the PI, Dr. Mark Gladwin from the NIH Clinical Center. Nationally, 142 subjects are expected to enroll over an 18 month period. Locally 20 subjects will be enrolled. The study population is comprised of persons with SS or S-Beta-thalassemia who present with VOC. 80 ppm inhaled NO will be given by face mask using a blinded version of the INOvent continuous flow, delivery system for 4 hours then 40 ppm for another 4 hours. It will be delivered with air and oxygen to an FIO2 of 24%. If required, patients in the NO group who remain in the hospital will continue to receive 800 ppm NO by inhalation at 6 mL/pulse/breath via nasal cannula using the INOpulse, pulsed flow, delivery system, for up to 72 hours total treatment time with 1 liter continuous oxygen if 60 pounds (27kg) or greater or 3 ml/pulse/breath if less than 60 pounds. Placebo is 100% grade 5 nitrogen gas delivered with oxygen at 24% by continuous flow for 8 hours. Patients who remain in the hospital will continue to receive it by nasal cannula at 6ml/pulse/breath at 800 ppm using the INO pulse delivery system with 1 liter continuous oxygen if 60 pounds or greater and 3 mL/pulse/breath if less than 60 pounds up to 72 hours total. Primary efficacy is time to resolution of VOC. Safety endpoints are incidence and types of reported SAE and proportion of patients withdrawn from study for safety issues. Primary analysis is a Wilcoxon ranked sum test or in case of censored times to crisis resolution the Gehan-Wilcoxon test to compare the difference in median duration of VOC.

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 该研究是一项由制药商发起的行业/NIH内部赞助的试验，涉及NIH和INO Therapeutics之间的合作研究和开发协议。 目的是确定吸入一氧化氮（NO）治疗镰状细胞病患者血管闭塞性疼痛危象（VOC）的安全性和有效性。 这是一项由INO Therapeutics申办的前瞻性、多中心、双盲、随机、安慰剂对照试验，研究由NIH临床中心的PI Mark Gladwin博士发起。在全国范围内，预计142例受试者将在18个月内入组。 当地将招募20名受试者。 研究人群由患有VOC的SS或S-β-地中海贫血患者组成。将使用盲态版本的INOvent连续流输送系统通过面罩给予80 ppm吸入NO 4小时，然后再给予40 ppm 4小时。它将与空气和氧气一起输送到24%的FIO 2。 如果需要，留在医院的NO组患者将继续通过鼻插管使用INOpulse脉冲流输送系统以6 mL/脉冲/呼吸吸入800 ppm NO，总治疗时间长达72小时，如果60磅（27 kg）或更大，则使用1升连续氧气，如果小于60磅，则使用3 ml/脉冲/呼吸。 安慰剂为100% 5级氮气，通过连续流输送24%氧气8小时。 留在医院的患者将继续使用INO脉冲输送系统通过鼻插管以6 ml/脉冲/呼吸的800 ppm剂量接受治疗，如果60磅或以上，则使用1升连续氧气，如果小于60磅，则使用3 mL/脉冲/呼吸，总计72小时。 主要疗效是VOC消退的时间。 安全性终点是报告的SAE的发生率和类型以及因安全性问题退出研究的患者比例。 主要分析是Wilcoxon秩和检验，或在危机解决时间删失的情况下，Gehan-Wilcoxon检验，以比较VOC中位持续时间的差异。