ACTHAR Therapy for Central Nervous System Sarcoidosis

Sarcoidosis Clinical Trial

Official title:

ACTHAR Therapy for Central Nervous System Sarcoidosis

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02920710
• Other study ID #: ACTHAR CNS
• Status: Withdrawn
• Phase: Phase 4
• Start date: February 1, 2019
• Est. completion date: November 2, 2020

Study information

• Verified date: November 2020
• Source: The Cleveland Clinic
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: November 2, 2020
• Est. primary completion date: November 2, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Sarcoidosis

Intervention

Drug:
• Repository Corticotropin Injection
Initial treatment with 80 units daily for ten days (induction phase) Maintenance treatment with 80 units twice weekly (maintenance phase)

Locations

Country	Name	City	State
United States	Cleveland Clinic Main Campus	Cleveland	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
The Cleveland Clinic	Mallinckrodt

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of patients with clinically significant improvement - successful glucocorticoid tapering.		12 Weeks
Secondary	Proportion of patients with clinically significant improvement - no need for escalation of other therapy.		12 Weeks