Ruxolitinib for Kohlmeier-Degos Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a drug called ruxolitinib for its potential to treat Kohlmeier-Degos Disease (K-D), a rare condition affecting the central nervous system (CNS). K-D can block small blood vessels in various organs, including the brain and spinal cord, and currently lacks effective treatments for its CNS effects. The trial is specifically designed for a single participant, a 58-year-old male with this condition. Over up to 26 weeks, he will take ruxolitinib tablets twice daily, with regular health check-ups and tests to monitor his response.
As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering the participant a chance to be among the first to receive this potentially groundbreaking treatment.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial doctors to get a clear answer.
Is there any evidence suggesting that ruxolitinib is likely to be safe for humans?
Research has shown that ruxolitinib is generally safe for use. Other studies have used it to treat conditions like myelofibrosis and polycythemia vera, with patients typically experiencing no major issues. For instance, reports indicate that when used as a cream for skin conditions, it does not cause significant side effects.
Some individuals might experience mild to moderate side effects, but serious problems are rare. This trial is in the early stages and focuses on assessing safety, so there is limited data on using ruxolitinib specifically for Kohlmeier-Degos disease affecting the central nervous system. However, its use in other conditions offers some reassurance about its safety.12345Why do researchers think this study treatment might be promising?
Researchers are excited about Ruxolitinib for treating Kohlmeier-Degos Disease because it works differently from current options. While most treatments focus on managing symptoms, Ruxolitinib is a JAK1/2 inhibitor, which targets specific pathways involved in inflammation and immune response. This targeted approach offers potential not just for symptom relief, but for addressing the underlying processes of the disease, making it a promising option for patients with limited effective treatments available.
What evidence suggests that ruxolitinib might be an effective treatment for Kohlmeier-Degos disease affecting the CNS?
Research has shown that ruxolitinib effectively treats various conditions by reducing inflammation and controlling the immune system. For patients with acute graft-versus-host disease, it has outperformed other treatments. In this trial, participants will receive ruxolitinib to evaluate its potential for treating Kohlmeier-Degos disease affecting the central nervous system (CNS). Although using ruxolitinib for this specific condition is new, its success in managing immune-related issues suggests it might reduce inflammation and improve symptoms. The drug has also treated skin conditions like eczema, providing relief and improving skin health. These findings offer hope that ruxolitinib might benefit those with Kohlmeier-Degos disease affecting the CNS.678910
Who Is on the Research Team?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participant receives ruxolitinib treatment, starting with 5 mg BID for 1 week, then 10 mg BID for 13-73 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Ruxolitinib
Ruxolitinib is already approved in United States, European Union for the following indications:
- Intermediate or high-risk myelofibrosis
- Polycythemia vera
- Steroid-refractory acute graft-versus-host disease
- Chronic graft-versus-host disease
- Vitiligo
- Intermediate or high-risk myelofibrosis
- Polycythemia vera
- Steroid-refractory acute graft-versus-host disease
- Chronic graft-versus-host disease
- Non-segmental vitiligo
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Heart, Lung, and Blood Institute (NHLBI)
Lead Sponsor