CNS SarcoidosisPhase 4NCT02298491

Treatment of CNS Sarcoidosis With H.P. Acthar Gel

A Phase 4 study testing H.P. Acthar Gel in people with CNS Sarcoidosis, run by University of Maryland, Baltimore at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Any age
Conditions the study is for: CNS Sarcoidosis
What is being tested?: H.P. Acthar Gel
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 4 participants
Where: 1 siteUnited States
Study dates: May 2016to Nov 2020
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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.

Who cannot join

None listed.

Trial locations and contacts

1 site in 1 country.

United States1 site

University of Maryland, Baltimore
Baltimore, Maryland, 21201

Other trials in United States

About this study

Official title: Clinical Biomarkers of Disease Activity and Treatment Responses in Patients With CNS Sarcoidosis Treated With H.P. Acthar Gel

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.

Read the 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.

Conditions: CNS Sarcoidosis
Being tested: H.P. Acthar Gel

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

H.P. Acthar GelDrug
Also called ACTH
80 IU subcutaneously daily for 10 days then followed by 80 IU subcutaneously three times per week through Month 12

Study groups in NCT02298491
Study group	Role	What they receive
H.P. Acthar Gel	Experimental	Participants will receive active treatment with H.P. Acthar Gel Receives: Drug: H.P. Acthar Gel

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 12 months (intention to treat)
Change in Total Number of New Lesions
Change in the total number of lesions assessed at 1 year
Measured: 1 year

Secondary outcomes (5)

Patient-Determined Disease Steps (PDDS)
A patient-reported measure of disability which consists of a scale with 9 levels of disability ranging from 0 to 8 (higher scores are associated with worse disability).
Measured: 12 months (intention to treat)
Montreal Cognitive Assessment (MoCA)
The MoCA is a 30-point test that screens for cognitive impairment. Scores range from 0 to 30, with higher scores associated with a better outcome.
Measured: 12 months
Symbol-Digit Modalities Test (SDMT)
Assesses cognitive function. The patient is given a set of symbols, and each symbol is paired with a number. The patient is given 90 seconds to write down the number that corresponds to each symbol.Scores range from 0 to 110. A higher score is associated with a better outcome.
Measured: 12 months (intention to treat)
Work Productivity and Activities Impairment -General Health (WPAI-GH)
The WPAI-GH consists of six questions: 1 = currently employed; 2 = hours missed due to health problems; 3 = hours missed for other reasons; 4 = hours actually worked; 5 = degree that the patient's health affected productivity while working (using a 0 to 10 Visual Analogue Scale (VAS)); 6 = degree health affected productivity in regular unpaid activities (VAS). The result for question 5 is used for the outcome measure, for which a higher number is associated with a worse outcome.
Measured: 12 months (intention to treat)
Beck Depression Inventory
The Beck Depression Inventory measures the severity of depression in adolescents and adults. It consists of 21 subscales, and the score for each individual subscale is added to give a total score of between 0 to 63. Total scores <= 10 are considered normal. Scores > 10 are considered abnormal, with higher scores above that cutoff being associated with a worse outcome.
Measured: 12 months (intent to treat)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

May 2016Nov 2020

First posted24 Nov 2014 (estimated)
Study startMay 2016
Primary completionNov 2020
Study completionNov 2020

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (11)Papers the sponsor has linked to this study.
Other trials for CNS SarcoidosisStudies of the same condition, open and under way.
More trials run by University of Maryland, BaltimoreThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 4 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02298491
Sponsor's protocol ID: HP-00062490
Status last verified: Apr 2026
First submitted: 14 Nov 2014
Last updated: 17 Apr 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

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.
PubMed 12172449
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.
PubMed 23034287
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.
PubMed 15081537
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.
PubMed 24751450
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.
PubMed 3347340
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.
PubMed 16432251
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.
PubMed 12588536
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.
PubMed 19539379
Shi C, Pamer EG. Monocyte recruitment during infection and inflammation. Nat Rev Immunol. 2011 Oct 10;11(11):762-74. doi: 10.1038/nri3070.
PubMed 21984070
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.
PubMed 19932381
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.
PubMed 3896208

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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All CNS Sarcoidosis trials

Study at a glance

From the registry record, last updated by the sponsor on 17 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Clinical Biomarkers of Disease Activity and Treatment Responses in Patients With CNS Sarcoidosis Treated With H.P. Acthar Gel
ClinicalTrials.gov ID: NCT02298491
Acronym / sponsor ID: HP-00062490
Conditions: CNS Sarcoidosis
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 4 participants (actual)
Lead sponsor: University of Maryland, BaltimoreUniversity, hospital or other
Collaborators: Mallinckrodt
Study start: May 2016
Primary completion: Nov 2020 (actual)
Study completion: Nov 2020 (actual)
First posted: 24 Nov 2014 (estimated)
Results: Posted 4 Feb 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
H.P. Acthar Gel	Experimental	Drug: H.P. Acthar GelParticipants will receive active treatment with H.P. Acthar Gel

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

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.

Key exclusion criteria (0 listed)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
H.P. Acthar GelAlso: ACTH80 IU subcutaneously daily for 10 days then followed by 80 IU subcutaneously three times per week through Month 12	Drug	H.P. Acthar Gel

Primary outcome

Karnofsky Performance Status (KPS) Score
Time frame: 12 months (intention to treat)
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.
Change in Total Number of New Lesions
Time frame: 1 year
Change in the total number of lesions assessed at 1 year

5 secondary outcomes

Patient-Determined Disease Steps (PDDS)
Time frame: 12 months (intention to treat)
Montreal Cognitive Assessment (MoCA)
Time frame: 12 months
Symbol-Digit Modalities Test (SDMT)
Time frame: 12 months (intention to treat)
Work Productivity and Activities Impairment -General Health (WPAI-GH)
Time frame: 12 months (intention to treat)
Beck Depression Inventory
Time frame: 12 months (intent to treat)

Results and safety

Results posted to the registry on 4 Feb 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
H.P. Acthar Gel	4	3

Primary outcome: Karnofsky Performance Status (KPS) Score

Time frame: 12 months (intention to treat). Mean (Standard Deviation). Unit: score on a scale

Measure	H.P. Acthar Gel Pre-treatment n = 3	H.P. Acthar Gel Post-treatment n = 3
Value	76.67 (15.28)	76.67 (5.77)

H.P. Acthar Gel Pre-treatment vs H.P. Acthar Gel Post-treatment: Mean Difference (Net): 0, p = 1.0, t-test, 2 sided

Population analysed: The population consisted of individuals with neurosarcoidosis before and after treatment with Acthar Gel

Primary outcome: Change in Total Number of New Lesions

Time frame: 1 year. Number. Unit: lesions

Measure	H.P. Acthar Gel n = 3
Value	4

H.P. Acthar Gel: lesion number increase or decrease: 0.25, p = 0.56, Sign test

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: 1 year. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
H.P. Acthar Gel	0 of 4 (0%)	0 of 4 (0%)	0 of 4

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 12172449Agostini 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.
Background: PubMed 23034287Arnason 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.
Background: PubMed 15081537Co 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.
Background: PubMed 24751450Judson 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.
Background: PubMed 3347340Miller 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.
Background: PubMed 16432251Miyara 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.
Background: PubMed 12588536Moller 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.
Background: PubMed 19539379Royal 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.
Background: PubMed 21984070Shi C, Pamer EG. Monocyte recruitment during infection and inflammation. Nat Rev Immunol. 2011 Oct 10;11(11):762-74. doi: 10.1038/nri3070.
Background: PubMed 19932381Stern 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.
Background: PubMed 3896208Stern 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.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Horea Rus principal investigator

University of Maryland, Baltimore

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing H.P. Acthar Gel in people with CNS Sarcoidosis, run by University of Maryland, Baltimore at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 4 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Karnofsky Performance Status (KPS) Score (time frame: 12 months (intention to treat)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02298491, which also lists 11 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Apr 2026.