Non-Invasive Treatment for Uveitis

葡萄膜炎的非侵入性治疗

• 批准号: 9060548
• 负责人: William I Higuchi
• 金额: $ 0.5万
• 依托单位: ACIONT, INC.
• 依托单位国家: 美国
• 财政年份: 2004
• 资助国家: 美国
• 起止时间: 2004-09-30 至 2016-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9060548
• 关键词: Acute; Adverse effects; American; Anterior; Back; Blindness; Chronic; Clinical Research; Clinical Trials; Cyclic GMP; Data; Devices; Dexamethasone; Disease; Dose; Drug Controls; Drug Delivery Systems; Drug Formulations; Drug Kinetics; Effectiveness; Ensure; Evaluation; Eye; Eye Part; Feedback; Funding; Goals; Guidelines; Health; Human; Hydrogels; Injection of therapeutic agent; Intermediate Uveitis; Lead; Marketing; Masks; Methods; Modality; Modeling; Monitor; Nurses; Oral; Oryctolagus cuniculus; Participant; Patient Education; Patients; Pharmaceutical Preparations; Phase; Phase III Clinical Trials; Physicians; Placebo Control; Posterior Uveitis; Posterior eyeball segment structure; Procedures; Progress Reports; Protocols documentation; Randomized; Regimen; Reporting; Research; Safety; Silicones; Small Business Innovation Research Grant; Solutions; System; Testing; Treatment Protocols; United States; Uveitis; Vision; Work; autoimmune uveitis; base; cost effective; design; diabetic; experience; eye center; implantation; improved; innovation; intravitreal injection; lens; macular edema; man; manufacturing scale-up; novel; novel strategies; patient safety; phase 2 study; pre-clinical; prototype; safety study; sodium phosphate; stability testing; standard of care; volunteer

DESCRIPTION (provided by applicant): The aim of this innovative proposal is to commercialize a non-invasive topical product that treats uveitis which is safe and easy to administer. Uveitis is the third leading cause of blindness in the United States. Heretofore, the primary treatment of intermediate and posterior uveitis has been either oral medications (with significant systemic side effects) or invasive local methods, such as an intravitreal injection (IVT), periocular injection or implantation of a sustained release drug delivery device into the eye. Thus, there is a great need to develop a superior drug delivery system in terms of safety, patient acceptance and efficacy for uveitis. The same product could be used to treat other indications such as diabetic macular edema, a substantial market involving more than 4 million Americans. Aciont has completed all necessary preclinical GLP safety, proof-of-concept work in uveitis and manufacturing of our dexamethasone sodium phosphate ("DSP") Visulex system needed to satisfy the FDA's requirements to test our proposed treatment in man. There are 3 specific aims for our research plan: " Aim1-Phase I Clinical Study (safety of treatment modality). Demonstrate that our new drug delivery applicator and DSP formulations are safe and well tolerated in man. " Aim2-Phase II Clinical Study (efficacy in uveitis). Demonstrate that our non-invasive DSP Visulex treatment is both safe and effective in treating uveitis. " Aim3-Optimize Visulex-P System Design and Manufacturability of Components and Formulations. Potentially, our approach can lead to a non-invasive drug delivery system that allows for a very simple treatment which can be performed by a nurse or paraprofessional in the doctor's office within a range of 5- 10 minutes. Such a system has the potential to curtail a significant portion of the acute sight threatening condition of severe uveitis in a single treatment. A long term goal could be to train patients to administer our treatment system by themselves.

描述（由申请人提供）：该创新提案的目的是将一种安全且易于给药的治疗葡萄膜炎的非侵入性局部产品商业化。葡萄膜炎是美国第三大致盲原因。因此，中间和后葡萄膜炎的主要治疗是口服药物（具有显著的全身副作用）或侵入性局部方法，例如玻璃体内注射（IVT）、眼周注射或将持续释放药物递送装置植入眼睛中。因此，非常需要开发一种在安全性、患者接受性和葡萄膜炎疗效方面优越的上级药物递送系统。同样的产品可以用于治疗其他适应症，如糖尿病黄斑水肿，这是一个涉及400多万美国人的巨大市场。Aciont已经完成了所有必要的临床前GLP安全性，葡萄膜炎的概念验证工作，以及生产我们的地塞米松磷酸钠（“DSP”）Visulex系统，以满足FDA对人体测试我们拟议治疗的要求。我们的研究计划有3个具体目标：“目标1-I期临床研究（治疗方式的安全性）。证明我们的新型药物输送涂抹器和DSP制剂在人体中是安全的，耐受性良好。“目标2-II期临床研究（葡萄膜炎疗效）。证明我们的非侵入性DSP Visulex治疗在治疗葡萄膜炎方面安全有效。“目标3-优化Visulex-P系统设计和组件及配方的可制造性。潜在地，我们的方法可以导致非侵入性药物递送系统，其允许非常简单的治疗，可以由护士或辅助专业人员在医生办公室在5- 10分钟的范围内进行。这样的系统具有在单次治疗中减少严重葡萄膜炎的严重视力威胁状况的显著部分的潜力。一个长期的目标可能是训练患者自己管理我们的治疗系统。