Non-Invasive Treatment for Uveitis

葡萄膜炎的非侵入性治疗

• 批准号: 8455734
• 负责人: William I Higuchi
• 金额: $ 107.39万
• 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 2015-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8455734
• 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; 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; public health relevance; 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使用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失明原因。迄今为止，中后葡萄膜炎的主要治疗方法是口服药物（具有显着的全身副作用）或侵入性局部方法，例如玻璃体内注射（IVT）、眼周注射或将缓释药物输送装置植入眼内。因此，非常需要开发一种在安全性、患者接受度和葡萄膜炎疗效方面优异的药物递送系统。同样的产品还可用于治疗其他适应症，例如糖尿病性黄斑水肿，这是一个涉及超过 400 万美国人的庞大市场。 Aciont 已完成所有必要的临床前 GLP 安全性、葡萄膜炎概念验证工作，以及生产我们的地塞米松磷酸钠 (“DSP”) Visulex 系统，以满足 FDA 测试我们提议的人体治疗方法的要求。我们的研究计划有 3 个具体目标：“Aim1-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 制剂对人体安全且耐受性良好。”Aim2-II 期临床研究（葡萄膜炎的疗效）。证明我们的非侵入性 DSP Visulex 治疗治疗葡萄膜炎既安全又有效。 “Aim3-优化 Visulex-P 系统设计以及组件和配方的可制造性。我们的方法有可能带来一种非侵入性药物输送系统，该系统允许非常简单的治疗，可由护士或专业人员在医生办公室在 5-10 分钟内完成。这样的系统有可能在单次治疗中减少严重葡萄膜炎的急性视力威胁状况的很大一部分。长期目标可能是到 培训患者自行管理我们的治疗系统。