Trial Outcomes & Findings for JAK/STAT Inhibition in CNS Kohlmeier-Degos Disease (NCT NCT05998395)

Last Updated: 2026-02-03

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

1 participants

Primary outcome timeframe

Baseline, up to 13 weeks

Results posted on

2026-02-03

Participant Flow

Participant milestones

Participant milestones
Measure	Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease Ruxolitinib at 5 mg twice a day (BID) for 1 week and then at 10 mg BID for 13-73 weeks and 1 week of 5 mg BID before stopping ruxolitinib.
Overall Study STARTED	1
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease Ruxolitinib at 5 mg twice a day (BID) for 1 week and then at 10 mg BID for 13-73 weeks and 1 week of 5 mg BID before stopping ruxolitinib.
Overall Study Physician Decision	1

Baseline Characteristics

JAK/STAT Inhibition in CNS Kohlmeier-Degos Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease n=1 Participants Ruxolitinib at 5 mg twice a day (BID) for 1 week and then at 10 mg BID for 13-73 weeks and 1 week of 5 mg BID before stopping ruxolitinib.
Age, Categorical <=18 years	0 Participants n=13 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=13 Participants
Age, Categorical >=65 years	0 Participants n=13 Participants
Sex: Female, Male Female	0 Participants n=13 Participants
Sex: Female, Male Male	1 Participants n=13 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=13 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=13 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=13 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=13 Participants
Race (NIH/OMB) Asian	0 Participants n=13 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=13 Participants
Race (NIH/OMB) Black or African American	0 Participants n=13 Participants
Race (NIH/OMB) White	1 Participants n=13 Participants
Race (NIH/OMB) More than one race	0 Participants n=13 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=13 Participants
Region of Enrollment United States	1 participants n=13 Participants

PRIMARY outcome

Timeframe: Baseline, up to 13 weeks

Outcome measures

Outcome measures
Measure	Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease n=1 Participants Ruxolitinib at 5 mg twice a day (BID) for 1 week and then at 10 mg BID for 13-73 weeks and 1 week of 5 mg BID before stopping ruxolitinib.
Number of Participants With Stable or no Lesions Developed Based on MRI Neurological Involvement of K-D Disease Following Ruloxitinib Stabilization of Lesions	1 Participants
Number of Participants With Stable or no Lesions Developed Based on MRI Neurological Involvement of K-D Disease Following Ruloxitinib Regression of Lesions	0 Participants
Number of Participants With Stable or no Lesions Developed Based on MRI Neurological Involvement of K-D Disease Following Ruloxitinib New Lesions	0 Participants

PRIMARY outcome

Timeframe: 13 weeks up to 72 weeks

Outcome measures

Outcome measures
Measure	Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease n=1 Participants Ruxolitinib at 5 mg twice a day (BID) for 1 week and then at 10 mg BID for 13-73 weeks and 1 week of 5 mg BID before stopping ruxolitinib.
Number of Participants With Stable or no Lesions Developed Based on MRI Neurological Involvement of K-D Disease Following Ruloxitinib Stabilization of Lesions	0 Participants
Number of Participants With Stable or no Lesions Developed Based on MRI Neurological Involvement of K-D Disease Following Ruloxitinib Regression of Lesions	0 Participants
Number of Participants With Stable or no Lesions Developed Based on MRI Neurological Involvement of K-D Disease Following Ruloxitinib New Lesions	1 Participants

Adverse Events

Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease n=1 participants at risk Ruxolitinib at 5 mg twice a day (BID) for 1 week and then at 10 mg BID for 13-73 weeks and 1 week of 5 mg BID before stopping ruxolitinib.
Skin and subcutaneous tissue disorders Worsening Sacral wound abscess	100.0% 1/1 • Number of events 1 • Up to 72 weeks
Blood and lymphatic system disorders Anemia	100.0% 1/1 • Number of events 1 • Up to 72 weeks
Infections and infestations Bacteremia	100.0% 1/1 • Number of events 1 • Up to 72 weeks
Infections and infestations Sepsis	100.0% 1/1 • Number of events 4 • Up to 72 weeks

Other adverse events

Other adverse events
Measure	Ruxolitinib in Participants With CNS Kohlmeier-Degos Disease n=1 participants at risk Ruxolitinib at 5 mg twice a day (BID) for 1 week and then at 10 mg BID for 13-73 weeks and 1 week of 5 mg BID before stopping ruxolitinib.
Psychiatric disorders Depression	100.0% 1/1 • Number of events 1 • Up to 72 weeks
General disorders Abnormal MRI	100.0% 1/1 • Number of events 1 • Up to 72 weeks

Additional Information

Cornelia Cudrici, M.D.

National Heart, Lung, and Blood Institute (NHLBI) Institute

Phone: 301.451.7070

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place