NCT02920710

ACTHAR Therapy for Central Nervous System Sarcoidosis

• Status: Withdrawn
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: The Cleveland Clinic · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

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

Detailed description

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.

Conditions

• Sarcoidosis

Interventions

Type	Name	Description
DRUG	Repository Corticotropin Injection	* Initial treatment with 80 units daily for ten days (induction phase) * Maintenance treatment with 80 units twice weekly (maintenance phase)

Timeline

• Start date: 2019-02-01
• Primary completion: 2020-11-02
• Completion: 2020-11-02
• First posted: 2016-09-30
• Last updated: 2020-11-20

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study