Adults 18 to 100, any sex, with Kohlmeier Degos Disease With Neurologic Involvement or Kohlmeier-Degos Disease. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Stable or no Lesions Developed Based on MRI Neurological Involvement of K-D Disease Following RuloxitinibPrimary· Baseline, up to 13 weeks
The primary endpoint is stability or regression of existing enhancing lesions or no development of new enhancing lesions in the brain and spine observed in MRI evaluation after 13 weeks and up to 73 weeks of ruloxitinib (10 mg BID) compared to pre-treatment MRI images.
Group
Value
95% CI
Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease
1
Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease
0
Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease
0
Number of Participants With Stable or no Lesions Developed Based on MRI Neurological Involvement of K-D Disease Following RuloxitinibPrimary· 13 weeks up to 72 weeks
The primary endpoint is stability or regression of existing enhancing lesions or no development of new enhancing lesions in the brain and spine observed in MRI evaluation after 13 weeks and up to 73 weeks of ruloxitinib (10 mg BID) compared to pre-treatment MRI images.
Group
Value
95% CI
Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease
0
Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease
0
Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease
1
Adverse events — posted to ClinicalTrials.gov
Time frame: Up to 72 weeks.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease
Background:
Kohlmeier-Degos (K-D) is a rare disease that leads to the inflammation and/or blockage of small blood vessels in many organs; these can include the skin, eyes (rare), small bowels, lungs, heart, and the brain and spinal cord (central nervous system, or CNS). There are no known effective treatments for K-D that affects the CNS.
Objective:
To test a drug (ruxolitinib) in a person with K-D affecting the CNS.
Eligibility:
This study is designed to treat 1 adult participant with K-D affecting the CNS.
Design:
The participant will be screened:
They will have a physical exam and blood tests.
They will have skin biopsies: Small samples of skin will be removed.
They will have a lumbar puncture: A needle will be inserted in his back to draw fluid from the space around the spinal cord.
They will have a magnetic resonance imaging (MRI) scan: they will lie on a table that slides into a tube to take pictures of their brain and spinal cord.
They will see a doctor who specializes in nerves.
Ruxolitinib is a tablet taken by mouth. The participant will take the drug twice a day for up to 26 weeks. The dosage may change over time.
The participant will have up to 7 clinic visits in 28 weeks. Each visit will be 1 to 3 days. MRI scans, biopsies, lumbar punctures, and other blood tests will be repeated on different visits. The participant may receive follow-up phone calls between visits. The participant will report any adverse effects. Unscheduled visits may be needed if new symptoms develop.
The last follow-up will be 4 weeks after the last dose of the study drug.
Publications & conference data
No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.
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Sponsor: as reported to ClinicalTrials.gov by National Heart, Lung, and Blood Institute (NHLBI)
Last refreshed: 3 February 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05998395.