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Trial · Interventional

CompletedPhase 4Results postedRegistry data

Treatment of CNS Sarcoidosis With H.P. Acthar Gel

NCTNCT02298491 (opens ClinicalTrials.gov in a new tab)Other idHP-00062490
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 4
Enrollment
4 participants (actual)
Sponsor
University of Maryland, Baltimore · Other
Design
Allocation not applicable · Open label (no masking)
Countries
1 country

United States

Start
May 2016
Primary completion
Nov 2020
Completion
Nov 2020
First posted
24 Nov 2014 (estimated)
Last update posted
17 Apr 2026
Conditions
CNS Sarcoidosis

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
201620172018201920202021
  1. Ph 4

    Treatment of CNS Sarcoidosis With H.P. Acthar Gel

    NCT02298491

    May 2016 to Nov 2020

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • H.P. Acthar GelDrug

Arms1

  • Experimental

    H.P. Acthar Gel

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

Primary outcomes2

Outcomes the sponsor listed as primary.

  1. Karnofsky Performance Status (KPS) Score

    Time frame · 12 months (intention to treat)

    Registry description

    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.

  2. Change in Total Number of New Lesions

    Time frame · 1 year

    Registry description

    Change in the total number of lesions assessed at 1 year

Secondary outcomes(5)

  1. Patient-Determined Disease Steps (PDDS)

    Time frame · 12 months (intention to treat)

    Registry description

    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).

  2. Montreal Cognitive Assessment (MoCA)

    Time frame · 12 months

    Registry description

    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.

  3. Symbol-Digit Modalities Test (SDMT)

    Time frame · 12 months (intention to treat)

    Registry description

    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.

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

    Time frame · 12 months (intention to treat)

    Registry description

    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.

  5. Beck Depression Inventory

    Time frame · 12 months (intent to treat)

    Registry description

    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.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· 12 months (intention to treat)

Karnofsky Performance Status (KPS) Score

GroupValue (score on a scale)Spread
H.P. Acthar Gel Pre-treatment76.6715.28
H.P. Acthar Gel Post-treatment76.675.77
Primary outcomeNumber· 1 year

Change in Total Number of New Lesions

GroupValue (lesions)
H.P. Acthar Gel4
Secondary and other outcome results(5)
Secondary outcomeMean· 12 months (intention to treat)

Patient-Determined Disease Steps (PDDS)

GroupValue (score on a scale)Spread
H.P. Acthar Gel Pre-treatment32.65
H.P. Acthar Gel Post-treatment2.672.31
Secondary outcomeMean· 12 months

Montreal Cognitive Assessment (MoCA)

GroupValue (score on a scale)Spread
H.P. Acthar Gel Pre-treatment26.333.79
H.P. Acthar Gel - Post-treatment26.333.51
Secondary outcomeMean· 12 months (intention to treat)

Symbol-Digit Modalities Test (SDMT)

GroupValue (score on a scale)Spread
H.P. Acthar Gel Pre-treatment34.520.51
H.P. Acthar Gel Post-treatment5819.80
Secondary outcomeMean· 12 months (intention to treat)

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

GroupValue (score on a scale)Spread
H.P. Acthar Gel Pre-treatment2.332.52
H.P. Acthar Gel Post-treatment3.332.89
Secondary outcomeMean· 12 months (intent to treat)

Beck Depression Inventory

GroupValue (score on a scale)Spread
H.P. Acthar Gel Pre-treatment62.65
H.P. Acthar Gel Post-treatment52

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
H.P. Acthar GelH.P. Acthar Gel: 80 IU subcutaneously daily for 10 days then followed by 80 IU subcutaneously three times per week through Month 120 / 40 / 40 / 4

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
Not stated
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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:

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