Ruxolitinib for Kohlmeier-Degos Disease

Overseen by Cornelia D Cudrici, M.D.

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

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

No Placebo Group

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial tests ruxolitinib, a pill that reduces inflammation, in a 58-year-old man with a rare disease affecting his brain and spinal cord. The drug aims to block certain proteins to reduce inflammation and slow the disease's progression. Ruxolitinib has been used successfully in treating various conditions and has shown promise in skin-related issues.

Research Team

Cornelia D Cudrici, M.D.

Principal Investigator

National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria

This trial is specifically for a single participant, a 58-year-old male with CNS Kohlmeier-Degos Disease. He must not have critically low blood cell counts, severe liver or kidney dysfunction, or any active life-threatening infections to be eligible.

Inclusion Criteria

I am a 58-year-old male with CNS Kohlmeier Degos Disease.

Exclusion Criteria

Your kidney function is very low, with an eGFR/CreatCr level less than 30 mL/min.

Your platelet count is less than 50,000 per microliter of blood.

Your white blood cell count is too low.

Treatment Details

Interventions

• Ruxolitinib (JAK Inhibitor)

Trial Overview The trial is testing the effectiveness of Ruxolitinib, an oral medication taken twice daily for up to 26 weeks in treating CNS Kohlmeier-Degos Disease. The patient will undergo various tests including MRI scans and biopsies throughout the study.

Participant Groups

Group I: Ruxolitinib Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

National Heart, Lung, and Blood Institute (NHLBI)

Lead Sponsor

Trials

3,987

Recruited

47,860,000+

Dr. Gary H. Gibbons

National Heart, Lung, and Blood Institute (NHLBI)

Chief Executive Officer since 2012

MD from Harvard Medical School

Dr. James P. Kiley

National Heart, Lung, and Blood Institute (NHLBI)

Chief Medical Officer since 2011

MD from University of California, San Francisco