Ruxolitinib for Kohlmeier-Degos Disease

CD
Overseen ByCornelia D Cudrici, M.D.
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 2 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

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.

Who Is on the Research Team?

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Cornelia D Cudrici, M.D.

Principal Investigator

National Heart, Lung, and Blood Institute (NHLBI)

Are You a Good Fit for This Trial?

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.
See 4 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks
1 visit (in-person)

Treatment

Participant receives ruxolitinib treatment, starting with 5 mg BID for 1 week, then 10 mg BID for 13-73 weeks

26-73 weeks
Up to 7 visits (in-person) over 28 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks
Follow-up phone calls

What Are the Treatments Tested in This Trial?

Interventions

  • Ruxolitinib
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.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: RuxolitinibExperimental Treatment1 Intervention

Ruxolitinib is already approved in United States, European Union for the following indications:

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Approved in United States as Jakafi for:
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Approved in European Union as Jakavi for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

National Heart, Lung, and Blood Institute (NHLBI)

Lead Sponsor

Trials
3,987
Recruited
47,860,000+
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