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CNS Sarcoidosis and Acthar Gel

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ClinicalTrials.gov Identifier: NCT02298491
Recruitment Status : Recruiting
First Posted : November 24, 2014
Last Update Posted : June 29, 2018
Sponsor:
Collaborator:
Mallinckrodt
Information provided by (Responsible Party):
Horea Rus, University of Maryland

Brief Summary:
The purpose of this study is to see if treatment with H.P. Acthar® Gel will result in the improvement and long-term stabilization of clinical and radiographic abnormalities that occur in patients with CNS sarcoidosis. In addition, it will also look at whether treatment will be also associated with improvement in measures of quality of life. The treatment of CNS sarcoidosis involves the use of either corticosteroids such as prednisone or potent immunosuppressive agents such as methotrexate, both which can induce severe long term side effects. The adverse effects of steroids may be avoided by treatment with adrenocorticotropic hormone (ACTH), which is available for patient use as H.P. Acthar® Gel. The efficacies of H.P. Acthar® Gel in the treatment of CNS sarcoidosis and the impact on quality of life have not been previously studied. In addition, little is known regarding the expression of immune markers in CNS sarcoidosis and the association of such markers with disease activity and response to treatment.

Condition or disease Intervention/treatment Phase
CNS Sarcoidosis Drug: H.P. Acthar Gel Phase 4

Detailed Description:
Sarcoidosis is a chronic and frequently progressive systemic disease that affects the central nervous system (CNS) in approximately 5% of patients. The hallmark of the disease is the development of chronic inflammation with formation of non-caseating granulomas that can involve the brain parenchyma and meninges and appear as contrast-enhancing mass lesions on magnetic resonance imaging. The granulomas are primarily comprised of proinflammatory T cells (Th1 cells and Th17 cells) and macrophages which accumulate during the early stages of granuloma formation. The inflammation that is generated by these cells is modulated by anti-inflammatory responses mediated by Th2 cells and regulatory T (Treg) cells that later appear and populate the outer regions of the granuloma. The presence of Treg cells are of particular interest since these cell are also detected in high numbers in peripheral blood and the immune suppression that results may underlie the occurrence of anergy in patients with the disease. The treatment of CNS sarcoidosis involves the use of either corticosteroids or potent immunosuppressive agents, both which can induce severe long-term side effects. The adverse effects of steroids may be avoided by treatment with adrenocorticotropic hormone (ACTH), which is available for patient use as H.P. Acthar® Gel. The efficacy of H.P. Acthar® Gel in the treatment of CNS sarcoidosis and the impact on quality of life have not been previously examined. In addition, little is known regarding the expression of immune markers in CNS sarcoidosis and the association of such markers with disease activity and response to treatment. These issues, therefore, will be explored in the context of this proposal.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Clinical Biomarkers of Disease Activity and Treatment Responses in Patients With CNS Sarcoidosis Treated With H.P. Acthar Gel
Study Start Date : May 2016
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Sarcoidosis

Arm Intervention/treatment
Experimental: H.P. Acthar Gel Drug: H.P. Acthar Gel
80 IU subcutaneously daily for 10 days then followed by 80 IU subcutaneously three times per week through Month 12
Other Name: ACTH




Primary Outcome Measures :
  1. Change in the total number of lesions [ Time Frame: 1, 3, 6, and 12 months ]
    Change in total number of lesions and in the number and size of enhancing lesions 1, 3, 6 months and 12 months relative to baseline


Secondary Outcome Measures :
  1. Quality of life measures [ Time Frame: 4 weeks, 6 months and 12 months ]
    Change in Patient Determined Disease Steps (PDDS), Montreal Cognitive Assessment (MoCA), Symbol-Digit Modalities Test (SDMT), Short Form -36 Health Survey (SF-36), Work Productivity and Activities Impairment -General Health (WPAI-GH) and Beck Depression Inventory-II (BDI-II) at 4 weeks, 6 months and 12 months relative to baseline

  2. Quality of Life Measure [ Time Frame: 6 months and 12 months ]
    Treatment Satisfaction Questionnaire for Medication (TSQM) scores at 4 weeks and change in TSQM scores at 6 months and 12 months versus baseline


Other Outcome Measures:
  1. Serum and cerebrospinal fluid Immune Markers [ Time Frame: 4 weeks, 6 months and 12 months ]
    Change in levels of serum and cerebrospinal fluid immune markers at 4 weeks, 6 months and 12 months versus baseline



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • A highly probable diagnosis of sarcoidosis, as determined using the World Association for Sarcoidosis and Other Granulomatous Disorders (WASOG) Sarcoidosis Organ Assessment Instrument (Judson et al., 2014), with involvement not limited to the central nervous system.
  • At the time of enrollment, a history of clinical deterioration based on the development of new symptoms or worsening previously present symptoms with confirmation by clinical examination and objective clinical testing.
  • If on steroids, on a stable dose of the medication for at least 3 months.

Exclusion Criteria:


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): NCT02298491


Contacts
Contact: Kerry Naunton, MD 410-328-1885 knaunton@som.umaryland.edu
Contact: Valerie Wells 410-328-5532 vyoung@som.umaryland.edu

Locations
United States, Maryland
University of Maryland, Baltimore Recruiting
Baltimore, Maryland, United States, 21201
Contact: Karen Callison    410-328-7602    kcallison@som.umaryland.edu   
Contact: Kerry Naunton    410-328-1885    knaunton@som.umaryland.edu   
Sponsors and Collaborators
University of Maryland
Mallinckrodt
Investigators
Principal Investigator: Horea Rus University of Maryland

Publications:

Responsible Party: Horea Rus, Professor of Neurology, University of Maryland
ClinicalTrials.gov Identifier: NCT02298491     History of Changes
Other Study ID Numbers: HP-00062490
First Posted: November 24, 2014    Key Record Dates
Last Update Posted: June 29, 2018
Last Verified: May 2018

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