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ACTHAR Therapy for Central Nervous System Sarcoidosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02920710
Recruitment Status : Withdrawn (Difficulty with recruitment)
First Posted : September 30, 2016
Last Update Posted : November 20, 2020
Sponsor:
Collaborator:
Mallinckrodt
Information provided by (Responsible Party):
The Cleveland Clinic

Study Description
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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

Condition or disease Intervention/treatment Phase
Sarcoidosis Drug: Repository Corticotropin Injection Phase 4

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: ACTHAR Therapy for Central Nervous System Sarcoidosis
Actual Study Start Date : February 1, 2019
Actual Primary Completion Date : November 2, 2020
Actual Study Completion Date : November 2, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Sarcoidosis

Arms and Interventions
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Arm Intervention/treatment
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.
 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


Outcome Measures
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Primary Outcome Measures :
  1. Proportion of patients with clinically significant improvement - successful glucocorticoid tapering. [ Time Frame: 12 Weeks ]

Secondary Outcome Measures :
  1. Proportion of patients with clinically significant improvement - no need for escalation of other therapy. [ Time Frame: 12 Weeks ]

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02920710


Locations
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United States, Ohio
Cleveland Clinic Main Campus
Cleveland, Ohio, United States, 44195
Sponsors and Collaborators
The Cleveland Clinic
Mallinckrodt
Investigators
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Principal Investigator: Daniel Culver, DO The Cleveland Clinic
More Information
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Responsible Party: The Cleveland Clinic
ClinicalTrials.gov Identifier: NCT02920710    
Other Study ID Numbers: ACTHAR CNS
First Posted: September 30, 2016    Key Record Dates
Last Update Posted: November 20, 2020
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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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
Additional relevant MeSH terms:
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Sarcoidosis
Lymphoproliferative Disorders
Lymphatic Diseases
Adrenocorticotropic Hormone
 Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs