ACTHAR Therapy for Central Nervous System Sarcoidosis
| Status: | Recruiting |
|---|---|
| Conditions: | Endocrine |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 3/2/2019 |
| Start Date: | February 1, 2019 |
| End Date: | December 2019 |
| Contact: | Daniel Culver, DO |
| Email: | culverd@ccf.org |
| Phone: | 216-444-6508 |
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
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
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.
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.
Inclusion Criteria:
- 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
- Seizures
Exclusion Criteria:
- 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.
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