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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02920710|
Recruitment Status : Not yet recruiting
First Posted : September 30, 2016
Last Update Posted : September 12, 2018
|Condition or disease||Intervention/treatment||Phase|
|Sarcoidosis||Drug: Repository Corticotropin Injection||Phase 4|
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 :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||ACTHAR Therapy for Central Nervous System Sarcoidosis|
|Estimated Study Start Date :||October 2018|
|Estimated Primary Completion Date :||July 2019|
|Estimated Study Completion Date :||December 2019|
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.
Drug: Repository Corticotropin Injection
- Proportion of patients with clinically significant improvement - successful glucocorticoid tapering. [ Time Frame: 12 Weeks ]
- Proportion of patients with clinically significant improvement - no need for escalation of other therapy. [ Time Frame: 12 Weeks ]
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): NCT02920710
|Contact: Daniel Culver, DOfirstname.lastname@example.org|
|United States, Ohio|
|Cleveland Clinic Main Campus||Not yet recruiting|
|Cleveland, Ohio, United States, 44195|
|Contact: Daniel Culver, DO 216-444-6508 email@example.com|
|Principal Investigator:||Daniel Culver, DO||The Cleveland Clinic|