ACTHAR Therapy for Central Nervous System Sarcoidosis

• ClinicalTrials.gov Identifier: NCT02920710
• Recruitment Status: Withdrawn
• First Posted: September 30, 2016
• Last Update Posted: November 20, 2020
• Sponsor: The Cleveland Clinic
• Collaborator: Mallinckrodt
• Information provided by (Responsible Party): The Cleveland Clinic

Tracking Information

• First Submitted Date: September 29, 2016
• First Posted Date: September 30, 2016
• Last Update Posted Date: November 20, 2020
• Actual Study Start Date: February 1, 2019
• Actual Primary Completion Date: November 2, 2020 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: September 29, 2016): Proportion of patients with clinically significant improvement - successful glucocorticoid tapering. [ Time Frame: 12 Weeks ]
• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures (submitted: September 29, 2016): Proportion of patients with clinically significant improvement - no need for escalation of other therapy. [ Time Frame: 12 Weeks ]
• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: ACTHAR Therapy for Central Nervous System Sarcoidosis
• Official Title: ACTHAR Therapy for Central Nervous System Sarcoidosis
• Brief 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.

• Study Type: Interventional
• Study Phase: Phase 4
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: 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)

Other Names:

• H.P. Acthar Gel
• ACTH Gel
• ACTH

Study Arms

Experimental: 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.

Intervention: Drug: Repository Corticotropin Injection

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Withdrawn
• Actual Enrollment (submitted: November 19, 2020): 0
• Original Estimated Enrollment (submitted: September 29, 2016): 20
• Actual Study Completion Date: November 2, 2020
• Actual Primary Completion Date: November 2, 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02920710
• Other Study ID Numbers: ACTHAR CNS
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: The Cleveland Clinic
• Original Responsible Party: Same as current
• Current Study Sponsor: The Cleveland Clinic
• Original Study Sponsor: Same as current
• Collaborators: Mallinckrodt

Investigators

• Principal Investigator: Daniel Culver, DO; The Cleveland Clinic

• PRS Account: The Cleveland Clinic
• Verification Date: November 2020