ACTHAR Therapy for Central Nervous System Sarcoidosis
试验速览
- 阶段
- 4 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Proportion of patients with clinically significant improvement - successful glucocorticoid tapering.
研究概览
简要总结
There is a need for a more reliable, expeditious therapy that can be used as an alternative to glucocorticoids in severe Central Nervous System (CNS) sarcoidosis. This study aims to provide evidence for effectiveness of ACTHAR gel in CNS sarcoidosis, and provide information about its safety and tolerability
详细描述
Central nervous system (CNS) involvement is one of the most severe manifestations of sarcoidosis. Sarcoidosis affecting the leptomeninges, spinal cord, or brain parenchyma portends a difficult course and frequently results in severe disability or death (1). Treatment of moderate and severe CNS sarcoidosis typically involves a combination of corticosteroids and cytotoxic agents such as methotrexate (2). Unfortunately, most response rates are reportedly only in the 29-38% range for corticosteroids alone, and the effects of cytotoxic agents in sarcoidosis require up to 6 months to occur. A typical scenario is that patients are treated for prolonged periods with high dose glucocorticoids with suboptimal effectiveness despite development of substantial toxicities. Some series report that cyclophosphamide or infliximab may be beneficial (3), but these approaches are limited by potentially severe toxicities, loss of effectiveness, or payor constraints.
. ACTHAR is a 39-amino acid peptide natural form of adrenocorticotropin hormone (ACTH) that was initially approved in 1952 by the FDA. It has since been approved for 19 indications including respiratory sarcoidosis, multiple sclerosis, and infantile spasms.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient with sarcoidosis as defined by ATS/ERS/WASOG (American Thoracic Society/European Thoracic Society/World Association for Sarcoidosis and Other Granulomatous Disorders)
- •Stable baseline immunosuppressive medications
- •Moderate to severe disease as defined by at least one of the following criteria:
- •Cranial nerve palsy
- •Neurologic deficits related to intraparenchymal brain, spinal cord and/or cauda equina involvement
- •Dural or leptomeningeal involvement of brain and/or spinal cord
- •Hydrocephalus
排除标准
- •Diagnosis of any underlying neurologic disorder that would potentially confound interpretation of the study results
- •Significant change in corticosteroid dose within the past 4 weeks, or other immunosuppressive medication within the past 6 months
- •Evidence of current serious infection, or a history of chronic or recurring infections.
- •Contraindication to high-dose corticosteroids (e.g. uncontrolled blood sugar).
- •Allergies to pig-derived proteins
- •Have a history of any opportunistic infection within 6 months prior to screening
- •History of malignancy.
研究组 & 干预措施
Experimental: H.P. Achtar Gel 80 U
H.P. Acthar Gel (repository corticotropin) Injection, 80 U daily for 10 days, then 80 U twice weekly for up to a total of 48 weeks on therapy.
干预措施: Repository Corticotropin Injection (Drug)
结局指标
主要结局
Proportion of patients with clinically significant improvement - successful glucocorticoid tapering.
时间窗: 12 Weeks
次要结局
- Proportion of patients with clinically significant improvement - no need for escalation of other therapy.(12 Weeks)