Phase IIa study of VBP15 for Duchenne muscular dystrophy

VBP15 治疗杜氏肌营养不良症的 IIa 期研究

• 批准号: 9047701
• 负责人: Paula R Clemens
• 金额: $ 149.53万
• 依托单位: REVERAGEN BIOPHARMA, INC.
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-02-15 至 2018-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9047701
• 关键词: 7 year old; Acute; Adult; Adverse effects; Age; Anti-Inflammatory Agents; Anti-inflammatory; Biological Markers; Body mass index; Characteristics; Chemistry; Childhood; Clinical; Clinical Trials; Data; Department of Defense; Development; Disease; Dose; Drug Kinetics; Duchenne muscular dystrophy; Enrollment; Essential Drugs; Formulation; Foundations; Funding; Future; Glucocorticoids; Goals; Government; Grant; Growth; Human; International; Leadership; Legal patent; Maps; Measures; Membrane; Methods; Monitor; Muscle; Muscular Dystrophies; Natural History; Neuromuscular Diseases; Neuromuscular research; Osteopenia; Outcome Measure; Pamphlets; Patients; Pharmaceutical Preparations; Pharmacodynamics; Pharmacologic Substance; Pharmacotherapy; Phase; Phase I Clinical Trials; Population; Positioning Attribute; Prednisone; Probability; Program Development; Proteomics; Protocols documentation; Quality of life; Research; Research Personnel; Risk Management; Safety; Sales; Seeds; Serum; Small Business Innovation Research Grant; Staging; Steroids; Structure; Testing; Therapeutics for Rare and Neglected Diseases; Time; Toxic effect; Toxicology; Transactivation; United States National Institutes of Health; Writing; biomarker panel; bone; boys; commercialization; comparative; deflazacort; drug development; improved; innovation; novel; peripheral blood; pharmacodynamic biomarker; pre-clinical; programs; public health relevance; research clinical testing; response; standard of care; tool; trial design; volunteer

 DESCRIPTION (provided by applicant): ReveraGen BioPharma is a clinical stage drug development company that is developing VBP15, a novel dissociative steroidal class drug. The initial indication for development is Duchenne muscular dystrophy, where VBP15 holds promise for retaining or increasing efficacy of glucocorticoids, while reducing side effects (bone fragilit, stunting of growth). VBP15 is currently in Phase I clinical trials in adult volunteers. This proposed Phase II SBIR research is to carry out a Phase IIa clinical trial in Duchenne muscular dystrophy boys, steroid naïve, ages 4-7 yrs. Aim 1 is to test four dose levels of VBP15 in a multiple ascending dose (MAD) trial design, with two weeks on drug. Patients completing the Phase IIa clinical trial will be offered enrollment into a six month extension study. The goal is t test pediatric pharmacokinetics, tolerability and safety in the Phase IIa clinical trial, and assessments of efficacy (time to stand velocity) and safety (change in body mass index) in the six month extension study to aid dose selection in the future Phase IIb registration trial. Aim 2 i to develop a pharmacodynamics biomarker panel that can assess safety and efficacy markers in acute time frames from peripheral blood. We have completed a natural history of DMD serum biomarkers using both SomaScan and proteomics discovery methods, and present preliminary data on glucocorticoid- associated efficacy and safety biomarkers. The Phase II SBIR research will compare VBP15 biomarkers to glucocorticoid biomarkers as a means of assessing comparative safety and potential efficacy.

 描述（由应用提供）：Reveragen Biopharma是一家临床阶段药物开发公司，正在开发VBP15，这是一种新型的分离性立体类药物。发育的最初指示是Duchenne肌肉营养不良，其中VBP15有望保持糖皮质激素的有效性或提高糖皮质激素的有效性，同时减少副作用（骨骼脆弱性，生长的发育迟缓）。 VBP15目前正在成人志愿者中进行I期临床试验。这项提出的II期SBIR研究是在4 - 7岁的Duchenne肌肉营养不良男孩中进行IIA期临床试验。目的1是在多次升级剂量（MAD）试验设计中测试四个剂量水平的VBP15，并使用两周的药物测试。完成IIA期临床试验的患者将入学六个月的扩展研究。目的是在IIA期临床试验中测试小儿药代动力学，耐受性和安全性，以及在六个月的扩展研究中评估效率（速度变化的时间）和安全性（体重变化），以帮助未来IIB阶段IIB注册试验的剂量选择。 AIM 2 I开发了一个药效生物标志物面板，该面板可以评估外周血急性时间范围内的安全性和有效性标记。我们已经使用SOMASCAN和蛋白质组学发现方法完成了DMD串行生物标志物的自然历史，并提供了有关糖皮质激素相关效率和安全生物标志物的初步数据。 II期SBIR研究将将VBP15生物标志物与糖皮质激素生物标志物进行比较，以评估比较安全性和潜在有效性。