A Reasonable Expectation of Effectiveness Clinical Trial of OKV-1001 for the Treatment of Primary (Non-Associative) Immune-Mediated Hemolytic Anemia in Dogs

OKV-1001治疗犬原发性（非相关性）免疫介导溶血性贫血临床试验有效性的合理预期

• 批准号: 10159258
• 负责人: ANDERSON WAYNE
• 金额: $ 24.96万
• 依托单位: OKAVA PHARMACEUTICALS, INC.
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-06-01 至 2024-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10159258
• 关键词: Reasonable; Expectation; Effectiveness; Clinical; Trial

Immune-mediated hemolytic anemia (IMHA) in dogs represents high unmet medical need. IMHA is considered to have a poor prognosis in dogs, with mortality rates of ~30%–40%. There are no FDA approved drugs. Well-controlled, prospective clinical studies for dogs with IMHA are limited, and leave treatment guidelines for IMHA in dogs poorly defined. The treatment, and management of symptoms, for canine IMHA is challenging. The mainstay immunosuppressive drug regimen for IMHA generally includes oral glucocorticoids, but individualized treatment plans based on response to treatment and severity of the disease are needed. There is a clinical need for immunosuppressive therapies that are safe and effective in treating IMHA. There is also a need for well-designed, prospective clinical studies that can be used to further inform the development of treatment guidelines. Mycophenolic acid (MPA) is an immune-modulating agent. Preliminary studies in dogs have demonstrated a potential role for MPA as a corticosteroid-sparing agent and as adjunctive or primary therapy in IMHA. OKV-1001, a novel extended-release MPA dosage form, is being developed to address limitations of the available human MPA preparations. The OKV-1001 extended-release formulation is designed to improve upon the existing MPA formulations by modulating the pharmacokinetic profile. The pharmacokinetic and pharmacodynamic properties observed in healthy dogs dosed with OKV-1001 are consistent with a once-daily formulation. The clinical performance of OKV-1001 for the treatment of IMHA will be studied in a randomized, double-blind, placebo-controlled, multicenter pilot study. This three-month study in sixty client-owned dogs, comparing the efficacy and safety of OKV-1001 to placebo, is an appropriate study design to support reasonable expectation of effectiveness of OKV-1001 as an adjunctive therapy for the treatment of newly-diagnosed idiopathic (non-associative) IMHA in dogs at least 12 months of age.

免疫介导性溶血性贫血(IMHA)在狗身上代表着高度未得到满足的医疗需求。 IMHA被认为对狗的预后很差，死亡率约为30%-40%。那里 都不是FDA批准的药物。对患有IMHA的狗进行了良好控制的前瞻性临床研究 有限，并离开治疗指南的狗IMHA定义不明确。 犬IMHA的治疗和症状管理是具有挑战性的。中流砥柱 IMHA的免疫抑制药物方案一般包括口服糖皮质激素，但 根据对治疗的反应和疾病的严重程度制定个性化的治疗计划 需要的。临床上需要安全有效的免疫抑制疗法。 治疗IMHA。还需要设计良好的前瞻性临床研究，这些研究可以 用于进一步通知治疗指南的制定。 霉酚酸是一种免疫调节剂。对狗的初步研究已经 证明了甲孕酮作为一种皮质类固醇节约剂和辅助剂的潜在作用。 IMHA的主要治疗方法。新型缓释甲孕酮剂型OKV-1001正在研制中 开发的目的是解决现有人用MPA制剂的局限性。OKV-1001 缓释制剂旨在通过以下方式改进现有的MPA制剂 调整药代动力学曲线。其药代动力学和药效学特性 在服用OKV-1001的健康狗身上观察到的结果与每天一次的配方一致。 OKV-1001治疗IMHA的临床性能将在 随机、双盲、安慰剂对照、多中心先导研究。这项为期三个月的研究 60只客户拥有的狗，将OKV-1001与安慰剂的疗效和安全性进行比较，是一种 适当的研究设计，以支持对OKV-1001作为一种 新诊断的特发性(非关联性)特发性(非关联性)IMHA的辅助治疗 至少12个月大的狗。