Oral N2O Therapy in Treating Acute Vaso-Occlusive Pain in Sickle Cell Disease
口服 N2O 疗法治疗镰状细胞病急性血管闭塞性疼痛
基本信息
- 批准号:10507724
- 负责人:
- 金额:$ 211.13万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2022
- 资助国家:美国
- 起止时间:2022-09-01 至 2024-08-31
- 项目状态:已结题
- 来源:
- 关键词:Abdominal colicAbsence of pain sensationAccident and Emergency departmentAcuteAcute PainAddressAnalgesicsAnestheticsAnimal ModelAnti-Anxiety AgentsBasic ScienceBiological MarkersBurn injuryCannulasCell DegranulationCell membraneChemistryClinicalClinical ResearchClinical SciencesClinical TrialsCoupledCyclic GMPDataDependenceDevelopmentDiseaseDoseDrug KineticsEquipmentErythrocytesEvaluationFDA approvedFormulationFractureFranceGasesGastrointestinal tract structureGeographyGlutamineGoalsHomeHospitalsHyperalgesiaImpairmentInflammationInhalationLaboratoriesLungMasksMedicalMethodsModalityMusNitrous OxideObstetric LaborObstructionOpioidOralOral AdministrationOrganOxygenPainPain managementPathologyPatientsPharmaceutical PreparationsPhasePhase II Clinical TrialsPreparationProceduresPropertyRattusRecurrenceReperfusion InjuryResearchResearch PersonnelResistanceRiskSafetySickle Cell AnemiaSickle HemoglobinSterile coveringsTechnologyTestingTherapeuticTissuesToxicologyabsorptionaqueousbasedrug developmentefficacy studyexperiencehealthy volunteerhydroxyureainnovationinnovative technologiesmanufacturabilitymanufacturing process developmentmast cellmouse modelnon-opioid analgesicnovelnovel therapeuticsopioid usepain modelpain reductionpediatric emergencypharmacokinetics and pharmacodynamicspreclinical efficacypreventvaso-occlusive crisisvaso-occlusive painventilation
项目摘要
The goal of the proposed project is to evaluate the potential of and develop HBI-201, a novel oral formulation of nitrous oxide (N2O), that enables the home use of N2O to treat pain associated with Vaso-Occlusive Crises (VOCs) in Sickle Cell Disease (SCD). The analgesic properties of N2O are well known, and N2O is an FDA- designated medical gas that is used for its analgesic, among other, properties. Importantly, current analgesic uses of N2O using inhaled gas include pain from SCD VOC in the emergency department (ED) in France, as well as in obstetric labor, acutely painful procedures such as fracture reduction, and in the management of the pain of ureteric colic. As a widely used, approved drug in the US, EU, and other geographies, the safety, tolerability, and efficacy of inhaled N2O (iN2O) has been demonstrated. However, barriers to home dosing of iN2O have prevented the development of an N2O therapeutic for home treatment of SCD VOC pain. Inhaled N2O gas is the approved modality for N2O. However, inhaled N2O is not a viable therapeutic option for home use due to poor dosing control, potential inhalation equipment issues and variability in patient ventilation and pulmonary absorption, the risk of inadvertent exposure due to leaking or incorrectly operated compressed gas cylinders, and patient resistance to gas therapies due to issues with mask and cannula use. HBI-201, a novel oral N2O drug product, enables home use of N2O in SCD and is being developed for the treatment of pain associated with VOCs in SCD. The administration of a defined dose of N2O delivered by oral administration of HBI-201 enables the further development of N2O as a therapeutic for home use while obviating the problems associated with iN2O. In this multi-PI application, we combine the expertise of the Simone and Belcher laboratories with Hillhurst’s experienced team and innovative technology and propose to build on compelling data to develop HBI-201 as a treatment for SCD VOC pain.
拟议项目的目标是评估和开发HBI-201的潜力,HBI-201是一种新型的一氧化二氮(N2 O)口服制剂,使家庭使用N2 O治疗与镰状细胞病(SCD)中血管闭塞性危机(VOC)相关的疼痛。N2 O的镇痛性质是众所周知的,并且N2 O是FDA指定的医用气体,其用于其镇痛等性质。重要的是,目前使用吸入气体的N2 O的镇痛用途包括法国急诊科(艾德)中SCD VOC引起的疼痛,以及产科分娩、急性疼痛手术(如骨折复位)和输尿管绞痛疼痛的管理。作为一种在美国、欧盟和其他地区广泛使用的获批药物,吸入性N2 O(iN 2 O)的安全性、耐受性和有效性已得到证实。然而,家庭给药iN 2 O的障碍阻碍了用于家庭治疗SCD VOC疼痛的N2 O治疗剂的开发。吸入N2 O气体是N2 O的批准方式。然而,由于剂量控制不良、潜在的吸入设备问题以及患者通气和肺吸收的可变性、由于泄漏或不正确操作压缩气瓶导致的意外暴露风险以及由于面罩和插管使用问题导致的患者对气体治疗的抵抗,吸入N2 O不是家庭使用的可行治疗选择。HBI-201是一种新型口服N2 O药物产品,可在SCD中家庭使用N2 O,目前正在开发用于治疗SCD中与VOC相关的疼痛。通过口服HBI-201递送的限定剂量的N2 O的施用使得能够进一步开发N2 O作为家庭使用的治疗剂,同时避免与iN 2 O相关的问题。在这个多PI应用中,我们将Simone和Belcher实验室的专业知识与Hillhurst经验丰富的团队和创新技术相结合,并建议以引人注目的数据为基础,开发HBI-201作为SCD VOC疼痛的治疗方法。
项目成果
期刊论文数量(0)
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JOHN D BELCHER其他文献
JOHN D BELCHER的其他文献
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{{ truncateString('JOHN D BELCHER', 18)}}的其他基金
Circulating Endothelial Cells and Microvesicles as Biomarkers for Gene Therapy in Sickle Cell Disease
循环内皮细胞和微泡作为镰状细胞病基因治疗的生物标志物
- 批准号:
10012207 - 财政年份:2019
- 资助金额:
$ 211.13万 - 项目类别:
Oral Carbon Monoxide Therapeutic to Prevent Vaso-Occlusive Crises in Sickle Cell Disease
口服一氧化碳治疗可预防镰状细胞病的血管闭塞危机
- 批准号:
9257461 - 财政年份:2016
- 资助金额:
$ 211.13万 - 项目类别:
HBI-002 to Prevent Vaso-Occlusive Crises in Sickle Cell Disease
HBI-002 可预防镰状细胞病的血管闭塞危机
- 批准号:
9789371 - 财政年份:2016
- 资助金额:
$ 211.13万 - 项目类别: