Recruiting

Phase 2

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

Sponsor:

National Heart, Lung, and Blood Institute (NHLBI)

Code:

NCT06923072

Conditions

Kohlmeier-Degos Disease

Malignant Atrophic Papulosis

Degos Disease

Papulosis, Malignant Atrophic

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

Baricitinib

Study Details

Brief summary:

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

Kohlmeier-Degos Disease

Malignant Atrophic Papulosis

Degos Disease

Papulosis, Malignant Atrophic

Study ID

NCT06923072

Start date

Feb 18, 2026

Status verified date

Jun 17, 2026

Completion date

Dec 15, 2030

Anticipated

Primary completion date

Dec 14, 2030

Anticipated

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

INCLUSION CRITERIA:

In order to be eligible to participate in this study, an individual must meet all of the following criteria:

Provision of signed and dated informed consent form by the subject or Legally Authorized Representative (LAR).
Stated willingness to comply with all study procedures and availability for the duration of the study.
Male or female, aged 18 or older.
Subjects diagnosed with systemic Degos disease, who manifest neurologic abnormalities observed clinically, radiologically or in abnormal laboratory findings.
Ability to take oral medication and be willing to adhere to the baricitinib regimen.
For female patients of reproductive potential, non-pregnant, non-breastfeeding: agree to use of highly effective contraception for the duration of the study and 30 days after the last dose.
Ability of subject or LAR to understand and the willingness to sign a written informed consent document.

EXCLUSION CRITERIA:

Active infection not responding to appropriate therapy
Hemoglobin <7 g/dL
Platelet counts < 50 K /mcL
Neutropenia (ANC <0.5 x k/mcL)
Lymphopenia (Absolute Lymphocyte Count \[ALC\] <0.2x k/mcL)
Liver function tests (LFTs > 2x time upper limit of normal)
Estimated Glomerular Filtration Rate (eGFR)/Creatinine (Cr < 30 mL/min)
Have experienced any of the following within 12 weeks of screening: VTE (DVT/pulmonary embolism \[PE\]), myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage III/IV heart failure.
Have had symptomatic herpes zoster infection within 12 weeks prior to a enrolling in the study .
Have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB.
Have evidence of active TB or latent TB
Have been exposed to a live vaccine within 12 weeks of baricitinib treatment or are expected to need/receive a live vaccine during the course of the study
No gadolinium based contrast agent exposure is permitted if eGFR < 30 mL/min/1.73m\^2 using the CKD-EPI equation measured within 5 days .
Breast feeding
Pregnancy
Uncontrolled malignancy

Study Design

Enrollment

12 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Participants with K(SqrRoot)(Delta)hlmeier-Degos Disease receiving Baricitinib

All participants will take baricitinib 4mg oral daily for 24 weeks.

Interventions

Baricitinib

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

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 ]
Number of participants with no new enhancing lesions in the brain and/or spine observed in MRI [ Time Frame: Baseline, Week 12, Week 24, Week 36 ]

Central Contacts and Locations

Central contacts

William R Whalen, C.R.N.P.

(301) 402-9841 william.whalen@nih.gov

Cornelia D Cudrici, M.D.

(240) 515-5540 cornelia.cudrici@nih.gov

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

NIH Clinical Center Office of Patient Recruitment (OPR)

800-411-1222 ccopr@nih.gov

More Information

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Last update posted

Jun 22, 2026

Last verified

Jun 17, 2026

Keywords

Degos Disease
Kohlmeier-Degos
Baricitinib
Neurological involvement of Degos Disease

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by National Heart, Lung, and Blood Institute (NHLBI) on 2026-06-22.