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
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?
1
Treatment groups
Experimental Treatment
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.
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
Citations
Efficacy, Safety, and Long-Term Disease Control of ...
Long-term intermittent use of ruxolitinib cream was well tolerated and provided disease control in adolescent patients with atopic dermatitis.
A treatment with 5-year overall survival data 1-4
At 3 years, survival probability was 70% for patients originally randomized to Jakafi and 61% for those originally randomized to placebo · OS was a prespecified ...
Novartis announces data showing Jakavi® (ruxolitinib) ...
Novartis announces data showing Jakavi® (ruxolitinib) more effective than best available therapy in acute graft-versus-host disease · Basel, April 22, 2020 ...
Ruxolitinib Cream 1.5%: A Review in Mild to Moderate ...
Additionally, the efficacy of ruxolitinib cream 1.5% was demonstrated for a 75% improvement from baseline in the Eczema Area and Severity Index ...
5.
hcplive.com
hcplive.com/view/pooled-phase-3-data-on-ruxolitinib-highlights-benefit-in-prurigo-nodularisPooled Phase 3 Data on Ruxolitinib Highlights Benefit in ...
Ruxolitinib cream demonstrated significant early and sustained improvements in prurigo nodularis symptoms through week 24 in phase 3 trials.
Safety Profile of Jakafi in aGVHD
Learn about the safety profile of Jakafi® (ruxolitinib) in the treatment of patients with steroid-refractory acute graft-versus-host disease (aGVHD).
JAK/STAT Inhibition in CNS Kohlmeier-Degos Disease
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 ...
Ruxolitinib: Uses, Interactions, Mechanism of Action
Ruxolitinib is a kinase inhibitor used to treat various types of myelofibrosis, polycythemia vera in patients who have not responded to or cannot tolerate ...
Real-World Use of Ruxolitinib Cream: Safety Analysis at 1 ...
Postmarketing safety data from the year following approval suggest ruxolitinib cream is generally well tolerated, without significant systemic ...
Pediatric Postmarketing Pharmacovigilance Review
The safety and effectiveness of Jakafi for treatment of steroid-refractory aGVHD has not been established in pediatric patients younger than 12 ...
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.