Treatment of CNS Sarcoidosis With H.P. Acthar Gel

NCT02298491 | Completed Phase 4 Results DMC

• 1 other identifier: HP-00062490
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

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.

Conditions

CNS Sarcoidosis

Outcome Measures

Primary Outcomes (2)

• Karnofsky Performance Status (KPS) Score

  The Karnofsky Performance Scale score allows patients to be classified as to their functional impairment. This can be used to compare the effectiveness of different therapies and to assess the prognosis in individual patients. The scale ranges from 0 to 100. Higher scores are associated with better outcomes.

  12 months (intention to treat)

• Change in Total Number of New Lesions

  Change in the total number of lesions assessed at 1 year

  1 year

Secondary Outcomes (5)

• Patient-Determined Disease Steps (PDDS)

  12 months (intention to treat)

• Montreal Cognitive Assessment (MoCA)

  12 months

• Symbol-Digit Modalities Test (SDMT)

  12 months (intention to treat)

• Work Productivity and Activities Impairment -General Health (WPAI-GH)

  12 months (intention to treat)

• Beck Depression Inventory

  12 months (intent to treat)

Study Arms (1)

H.P. Acthar Gel

EXPERIMENTAL

Participants will receive active treatment with H.P. Acthar Gel

Drug: H.P. Acthar Gel

Interventions

H.P. Acthar Gel DRUG

80 IU subcutaneously daily for 10 days then followed by 80 IU subcutaneously three times per week through Month 12

Also known as: ACTH

H.P. Acthar Gel

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

Sponsors & Collaborators

• University of Maryland, Baltimore: lead
• Mallinckrodt: collaborator

Study Sites (1)

University of Maryland, Baltimore

Baltimore, Maryland, 21201, United States

Location

Related Publications (11)

• Agostini C, Meneghin A, Semenzato G. T-lymphocytes and cytokines in sarcoidosis. Curr Opin Pulm Med. 2002 Sep;8(5):435-40. doi: 10.1097/00063198-200209000-00016.

  PMID: 12172449 BACKGROUND

• Arnason BG, Berkovich R, Catania A, Lisak RP, Zaidi M. Mechanisms of action of adrenocorticotropic hormone and other melanocortins relevant to the clinical management of patients with multiple sclerosis. Mult Scler. 2013 Feb;19(2):130-6. doi: 10.1177/1352458512458844. Epub 2012 Oct 3.

  PMID: 23034287 BACKGROUND

• Co DO, Hogan LH, Il-Kim S, Sandor M. T cell contributions to the different phases of granuloma formation. Immunol Lett. 2004 Mar 29;92(1-2):135-42. doi: 10.1016/j.imlet.2003.11.023.

  PMID: 15081537 BACKGROUND

• Judson MA, Costabel U, Drent M, Wells A, Maier L, Koth L, Shigemitsu H, Culver DA, Gelfand J, Valeyre D, Sweiss N, Crouser E, Morgenthau AS, Lower EE, Azuma A, Ishihara M, Morimoto S, Tetsuo Yamaguchi T, Shijubo N, Grutters JC, Rosenbach M, Li HP, Rottoli P, Inoue Y, Prasse A, Baughman RP, Organ Assessment Instrument Investigators TW. The WASOG Sarcoidosis Organ Assessment Instrument: An update of a previous clinical tool. Sarcoidosis Vasc Diffuse Lung Dis. 2014 Apr 18;31(1):19-27.

  PMID: 24751450 BACKGROUND

• Miller DH, Kendall BE, Barter S, Johnson G, MacManus DG, Logsdail SJ, Ormerod IE, McDonald WI. Magnetic resonance imaging in central nervous system sarcoidosis. Neurology. 1988 Mar;38(3):378-83. doi: 10.1212/wnl.38.3.378.

  PMID: 3347340 BACKGROUND

• Miyara M, Amoura Z, Parizot C, Badoual C, Dorgham K, Trad S, Kambouchner M, Valeyre D, Chapelon-Abric C, Debre P, Piette JC, Gorochov G. The immune paradox of sarcoidosis and regulatory T cells. J Exp Med. 2006 Feb 20;203(2):359-70. doi: 10.1084/jem.20050648. Epub 2006 Jan 23.

  PMID: 16432251 BACKGROUND

• Moller DR. Treatment of sarcoidosis -- from a basic science point of view. J Intern Med. 2003 Jan;253(1):31-40. doi: 10.1046/j.1365-2796.2003.01075.x.

  PMID: 12588536 BACKGROUND

• Royal W 3rd, Mia Y, Li H, Naunton K. Peripheral blood regulatory T cell measurements correlate with serum vitamin D levels in patients with multiple sclerosis. J Neuroimmunol. 2009 Aug 18;213(1-2):135-41. doi: 10.1016/j.jneuroim.2009.05.012. Epub 2009 Jun 17.

  PMID: 19539379 BACKGROUND

• Shi C, Pamer EG. Monocyte recruitment during infection and inflammation. Nat Rev Immunol. 2011 Oct 10;11(11):762-74. doi: 10.1038/nri3070.

  PMID: 21984070 BACKGROUND

• Stern BJ, Aksamit A, Clifford D, Scott TF; Neurosarcoidosis Study Group. Neurologic presentations of sarcoidosis. Neurol Clin. 2010 Feb;28(1):185-98. doi: 10.1016/j.ncl.2009.09.012.

  PMID: 19932381 BACKGROUND

• Stern BJ, Krumholz A, Johns C, Scott P, Nissim J. Sarcoidosis and its neurological manifestations. Arch Neurol. 1985 Sep;42(9):909-17. doi: 10.1001/archneur.1985.04060080095022.

  PMID: 3896208 BACKGROUND

MeSH Terms

Interventions

Adrenocorticotropic Hormone

Intervention Hierarchy (Ancestors)

Melanocortins; Pro-Opiomelanocortin; Hypothalamic Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Pituitary Hormones, Anterior; Pituitary Hormones; Neuropeptides; Peptides; Amino Acids, Peptides, and Proteins; Nerve Tissue Proteins; Proteins

Results Point of Contact

• Title: Walter Royal, III, MD
• Organization: Morehouse School of Medicine

wroyal@msm.edu

404-756-5222

Study Officials

• Horea Rus

  University of Maryland, Baltimore

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Neurology

Study Record Dates

• First Submitted: November 14, 2014
• First Posted: November 24, 2014
• Study Start: May 1, 2016
• Primary Completion: November 1, 2020
• Study Completion: November 1, 2020
• Last Updated: April 17, 2026
• Results First Posted: February 4, 2021

Record last verified: 2026-04

Locations

US (1)