Phase 4
N=4
Treatment of CNS Sarcoidosis With H.P. Acthar Gel
CNS Sarcoidosis
Bottom Line
View on ClinicalTrials.gov: NCT02298491 ↗Enrolled (actual)
4
Serious AEs
0.0%
Results posted
Feb 2021
Primary outcome: Primary: Karnofsky Performance Status (KPS) Score — 76.67; 76.67 score on a scale — p=1.0
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- H.P. Acthar Gel (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- University of Maryland, Baltimore
- Primary completion
- Nov 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Karnofsky Performance Status (KPS) Score |
76.67; 76.67 | 1.0 |
| PRIMARY Change in Total Number of New Lesions |
4 | 0.56 |
| SECONDARY Patient-Determined Disease Steps (PDDS) |
3; 2.67 | 0.88 |
| SECONDARY Montreal Cognitive Assessment (MoCA) |
26.33; 26.33 | 1.0 |
| SECONDARY Symbol-Digit Modalities Test (SDMT) |
34.5; 58 | 0.88 |
| SECONDARY Work Productivity and Activities Impairment -General Health (WPAI-GH) |
2.33; 3.33 | 0.34 |
| SECONDARY Beck Depression Inventory |
6; 5 | 0.31 |
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.
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT02298491). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.