A Phase 2 interventional study of Baricitinib in Kohlmeier-Degos Disease, Malignant Atrophic Papulosis and Degos Disease, sponsored by National Heart, Lung, and Blood Institute (NHLBI). Recruiting at 1 site in United States. Open to participants aged 18 Years to 99 Years. Per ClinicalTrials.gov, last updated 2026-09-18.
Sponsored by National Heart, Lung, and Blood Institute (NHLBI) · Phase 2, Interventional, and Treatment
Background:
Kohlmeier-Degos (KD) is a rare disease that causes inflammation and blood clots, leading to blockages in small blood vessels. These blockages can result in K-D lesions throughout the body, affecting the skin, lungs, heart, spinal cord, and brain. KD can be fatal. No treatment exists for this disease.
Objective:
To test a study drug (baricitinib) in people with brain and spine lesions caused by KD disease. Baricitinib is FDA approved to treat other disorders but has not yet been tried in people with KD.
Eligibility:
People aged 18 years or older with KD-related lesions in the brain and spine.
Design:
Participants will be screened; they will have a physical exam with blood tests. They will also have a baseline visit that may include multiple tests, such as imaging scans of the brain and spine; a lumbar puncture to collect fluid from the spinal canal; and a meeting with a neurologist. They will fill out a questionnaire about their health. They will continue to take their normal medications throughout the study.
Baricitinib is a tablet taken by mouth. Participants will remain on their normal medications for 12 weeks after their baseline visit. Then they will also take the study drug once a day at home for 24 weeks.
Participants will have clinic visits every few weeks for up to 40 weeks. Some visits may take 1 to 4 days. Baseline tests will be repeated 3 more times during study visits. Other visits will require only blood tests; these may be done by local labs that will send the samples to NIH; 2 visits may be done via telehealth....
Study Description:
This phase II study will provide off-label baricitinib treatment in patients with Kohlmeier Degos disease (K-D) with neurologic involvement. We will perform a baseline research evaluation at the time of enrollment and follow each patient for 12 weeks of background therapy (defined as medications taken by the subject for management of Degos symptoms) followed by 24 weeks of baricitinib treatment (4 mg daily) in addition to background therapy followed by 4 additional weeks of background therapy with final safety assessment at 40 weeks. We will repeat research evaluations at the end of weeks 12, 24, and 36. We will compare the disease progression between weeks 1 through 12 to weeks 13 through 24 as well as weeks 13 through 36. We hypothesize that baricitinib, which targets type I interferon (IFN) and IFN-g signaling, will attenuate various neurological manifestations of K-D that are observed clinically, radiologically or in abnormal laboratory findings in our KD patients. This will help reduce IFN signaling in a manner that may slow or halt the disease progression as measured by the endpoints established below.
Objectives:
Primary Objective:
To test whether baricitinib delays progression of neuroradiological manifestations in patients with neurological involvement of K-D disease.
Secondary Objectives:
To test whether baricitinib treatment improves patient-reported outcomes.
Exploratory Objectives:
Endpoints:
Exploratory endpoints of this study will be clinical and potential surrogate biomarker efficacy data, including:
National Heart, Lung, and Blood Institute (NHLBI) is the lead sponsor of 1,117 studies on the registry; 71 are open to participants now.
Of its 57 completed or terminated interventional studies of FDA-regulated products, 49 (86%) have results posted.
Counted across the registry records on this site, refreshed daily.
In order to be eligible to participate in this study, an individual must meet all of the following criteria:
EXCLUSION CRITERIA:
All participants will take baricitinib 4mg oral daily for 24 weeks.
Drug: Baricitinib
Participants will be instructed to take baricitinib 4mg oral daily for 24 weeks (with or without food)
Number of participants with stability of existing enhancing lesions in the brain and/or spine observed in MRI
Stability of existing enhancing lesions in the brain and/or spine observed in MRI after 12 or 24 weeks of baricitinib treatment (4 mg daily) as compared to MRI images after 12 weeks of background therapy only.
Time frame: Baseline, Week 12, Week 24, Week 36
Number of participants with no new enhancing lesions in the brain and/or spine observed in MRI
Lack of development of new enhancing lesions in the brain and/or spine observed in MRI after 12 or 24 weeks of baricitinib treatment (4 mg daily) as compared to MRI images after 12 weeks of background therapy only.
Time frame: Baseline, Week 12, Week 24, Week 36
Change in health outcome questionnaire, Short Form-36 (SF-36)
Compare the change in health outcome questionnaire, Short Form-36 (SF-36), over 12 and 24 weeks of baricitinib treatment as compared to 12 weeks of background therapy. This 36-item, patient-reported survey assesses quality of life. It consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability i.e., a score of zero is equivalent to maximum disability and a score of 100 is equivalent to no disability.
Time frame: Baseline, Week 12, Week 16, Week 24, Week 36, and up to Week 40
Neurological symptoms using CTCAE
Compare the change of neurological symptoms using CTCAE over 12 and 24 weeks of baricitinib treatment as compared to 12 weeks of background therapy.
Time frame: Up to 40 weeks
Plan to share: Yes — All IPD that underlie results in a publication
Supporting information: Study protocol, Sap, Icf
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Malignant Atrophic Papulosis
National Heart, Lung, and Blood Institute (NHLBI)