Actively Recruiting
Baricitinib in the Treatment of Kohlmeier-Degos Disease in Patients With Neurological Involvement
Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-04-03
12
Participants Needed
1
Research Sites
251 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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....
CONDITIONS
Official Title
Baricitinib in the Treatment of Kohlmeier-Degos Disease in Patients With Neurological Involvement
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent by participant or legal representative
- Willingness to comply with study procedures and available for study duration
- Male or female aged 18 years or older
- Diagnosed with systemic Degos disease showing neurologic abnormalities clinically, on imaging, or by lab tests
- Ability to take oral medication and willingness to adhere to baricitinib regimen
- For females of reproductive potential: not pregnant or breastfeeding and agree to use effective contraception during and 30 days after study
- Ability to understand and sign informed consent
You will not qualify if you...
- Active infection not responding to treatment
- Hemoglobin less than 7 g/dL
- Platelet count less than 50,000 per mcL
- Neutropenia with ANC less than 0.5 x 1000/mcL
- Lymphopenia with absolute lymphocyte count less than 0.2 x 1000/mcL
- Liver function tests more than twice the upper limit of normal
- Kidney function (eGFR) less than 30 mL/min
- History of venous thromboembolism, heart attack, unstable heart disease, stroke, or severe heart failure within 12 weeks
- Symptomatic herpes zoster infection within 12 weeks before enrollment
- Household contact with active tuberculosis without proper prophylaxis
- Active or latent tuberculosis
- Exposure to live vaccines within 12 weeks before or during the study
- Use of gadolinium contrast agents with eGFR under 30 mL/min/1.73m² within 5 days
- Breastfeeding
- Pregnancy
- Uncontrolled cancer
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
W
William R Whalen, C.R.N.P.
CONTACT
C
Cornelia D Cudrici, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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