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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Recruitment Status : Withdrawn (Difficulty with recruitment)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

Tracking Information
First Submitted Date  ICMJE September 29, 2016
First Posted Date  ICMJE September 30, 2016
Last Update Posted Date November 20, 2020
Actual Study Start Date  ICMJE February 1, 2019
Actual Primary Completion Date November 2, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 29, 2016)
Proportion of patients with clinically significant improvement - successful glucocorticoid tapering. [ Time Frame: 12 Weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (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  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE ACTHAR Therapy for Central Nervous System Sarcoidosis
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Sarcoidosis
Intervention  ICMJE 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  ICMJE 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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: November 19, 2020)
0
Original Estimated Enrollment  ICMJE
 (submitted: September 29, 2016)
20
Actual Study Completion Date  ICMJE November 2, 2020
Actual Primary Completion Date November 2, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02920710
Other Study ID Numbers  ICMJE 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  ICMJE
Plan to Share IPD: Undecided
Current Responsible Party The Cleveland Clinic
Original Responsible Party Same as current
Current Study Sponsor  ICMJE The Cleveland Clinic
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Mallinckrodt
Investigators  ICMJE
Principal Investigator: Daniel Culver, DO The Cleveland Clinic
PRS Account The Cleveland Clinic
Verification Date November 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP