Preclinical Development of Tacrolimus for Radiation Cystitis

他克莫司治疗放射性膀胱炎的临床前开发

基本信息

批准号：
8715380
负责人：
JONATHAN H KAUFMAN
金额：
$ 22.22万
依托单位：
LIPELLA PHARMACEUTICALS, INC.
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：
2014-05-12 至 2015-04-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8715380
关键词：
Basic Science Biological Availability Bladder Canis familiaris Caring Cessation of life Clinical Clinical Research Collaborations Cyclophosphamide Cystitis Data Development Dinoprostone Dose Drug Formulations Drug Kinetics Dysuria Foundations Funding Goals Hematuria Hemorrhage Histopathology Hospitals Immunosuppressive Agents Incidence Inflammatory Intellectual Property Interleukin-2 Intravenous Intravesical Administration Kidney Kidney Function Tests Lead Letters Life Liposomes Marketing Medical Modeling Oral Orphan Patients Pelvic Cancer Pharmaceutical Preparations Pharmacologic Substance Phase Property Rights Quality of life Radiation Radiation therapy Rare Diseases Rattus Reaction Research Research Proposals Research Support Rights Route Safety Small Business Innovation Research Grant Staining method Stains Tacrolimus Testing Therapeutic Therapeutics for Rare and Neglected Diseases Tissues Toxic effect Toxicology United States National Institutes of Health Universities Urinary tract Urine Urothelium Woman Work commercialization effective therapy follow-up human WFDC2 protein innovation intravesical lower urinary tract symptoms lymph nodes medical schools men novel phase 1 study pre-clinical programs public health relevance research study urologic

项目摘要

DESCRIPTION (provided by applicant): This project, entitied "Preclinical Development of Tracrolimus for Radiation Cystitis" will fund key experiments to advance the commercial development of LP-10. LP-10 is a liposomal tacrolimus formulation for local (topical) intravesical administration to the urinary bladder. The formulation provides active drug levels in the bladder with significantly reduced systemic levels. Radiation cystitis is a rare disease defined by lower urinary tract symptoms that include dysuria, hematuria, and hemorrhage. There are currently no approved therapies to treat RC, which can severely degrade a patient's quality of life, require long-term follow-up treatment, and, in some patients, lead to death. This project is public-private collaboration between Lipella Pharmaceuticals Inc., the University of Pittsburgh School of Medicine, and William Beaumont Hospital. Lipella owns intellectual property rights to the LP-10 formulation as well as rights associated with its recent receipt of orphan designation from the FDA. The FDA has also provided Lipella with pre-clinical requirements for IND consideration of LP-10. These requirements constitute the studies described in this proposal. The scope of work includes further pharmaceutical and pharmacological characterization of LP-10 (Phase I) and IND-enabling studies (Phase II). This research is translational, and bridges basic research to clinical development. Progress in this direction will directly support our ultimate goal of developing a safe and effective therapy ready for commercialization.

描述（由申请人提供）：该项目赋予“辐射膀胱炎的临床前开发”，将资助关键实验，以推动LP-10的商业发展。 LP-10是一种脂质体他克莫司，用于用于泌尿膀胱的局部（局部）静脉内给药。该配方在膀胱中提供了活跃的药物水平，并具有明显降低的全身水平。辐射膀胱炎是一种罕见的疾病，其定义为较低的尿路症状，包括排尿症，血尿和出血。目前尚无批准的治疗RC的疗法，可以严重降低患者的生活质量，需要长期随访治疗，在某些患者中，导致死亡。这个项目是公私的 Lipella Pharmaceuticals Inc.，匹兹堡大学医学院和William Beaumont医院之间的合作。 Lipella拥有LP-10配方的知识产权，以及与FDA最近收到孤儿指定相关的权利。 FDA还为Lipella提供了临床前的要求，以考虑LP-10。这些要求构成了本提案中描述的研究。工作范围包括LP-10（I阶段）和辅助研究（II期）的进一步药物和药理表征。这项研究是转化的，并将基础研究桥梁融入了临床发展。在这个方向上的进展将直接支持我们开发准备商业化的安全有效疗法的最终目标。