Baricitinib in the Treatment of Kohlmeier-Degos Disease in Patients With Neurological Involvement

Conditions

• Degos Disease
• Kohlmeier-Degos Disease
• Malignant Atrophic Papulosis
• Papulosis, Malignant Atrophic

Eligibility Criteria

Sex

ALL

Age

18 Years - 99 Years

Healthy Volunteers

Not accepted

Interventions

• Baricitinib — DRUG
  Participants will be instructed to take baricitinib 4mg oral daily for 24 weeks (with or without food)

Study Details

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....

Key Dates

Start date

Feb 18, 2026

Status verified

Apr 2026

Primary completion

Dec 14, 2030

Completion

Dec 15, 2030

Study Design

Enrollment

12 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Participants with K(SqrRoot)(Delta)hlmeier-Degos Disease receiving Baricitinib
  All participants will take baricitinib 4mg oral daily for 24 weeks.

Primary Outcome Measure

Number of participants with stability of existing enhancing lesions in the brain and/or spine observed in MRI [ Time Frame: Baseline, Week 12, Week 24, Week 36 ]

Central Contacts

• William R Whalen, C.R.N.P.
  (301) 402-9841
  [email protected]
• Cornelia D Cudrici, M.D.
  (240) 515-5540
  [email protected]

Locations (1)

Find similar trials in Bethesda, MD