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JAK Inhibitor
Ruxolitinib for Kohlmeier-Degos Disease
Phase 1 & 2
Recruiting
Led By Cornelia D Cudrici, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 6 months
Awards & highlights
Study Summary
This trial tests the drug ruxolitinib to treat a rare disease, Kohlmeier-Degos, that affects the brain and spinal cord. It involves physical exams, skin biopsies, MRI scans, and lumbar punctures. The participant will take the drug for up to 26 weeks and have 7 clinic visits.
Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
Potential side effects of Ruxolitinib may include changes in blood cell counts leading to increased infection risk, bleeding issues, liver function alterations, and other organ-related inflammations.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 6 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~6 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
To test the hypothesis that JAK/STAT inhibition by ruxolitinib will delay progression of neuroradiological manifestations of our one patient with neurological involvement of K-D disease.
Side effects data
From 2020 Phase 3 trial • 149 Patients • NCT0203803633%
Anaemia
19%
Hypertension
17%
Nasopharyngitis
16%
Weight increased
14%
Herpes zoster
14%
Constipation
14%
Abdominal pain
14%
Headache
12%
Pruritus
12%
Back pain
12%
Epistaxis
12%
Pyrexia
12%
Dizziness
10%
Asthenia
10%
Fatigue
10%
Cough
10%
Oedema peripheral
10%
Arthralgia
9%
Thrombocytosis
9%
Upper respiratory tract infection
9%
Hypercholesterolaemia
7%
Dyslipidaemia
7%
Pain in extremity
7%
Haematoma
7%
Abdominal discomfort
7%
Diarrhoea
7%
Dyspepsia
7%
Vomiting
7%
Blood lactate dehydrogenase increased
7%
Memory impairment
7%
Dyspnoea
5%
Tinnitus
5%
Osteoarthritis
5%
Leukocytosis
5%
Thrombocytopenia
5%
Flatulence
5%
Nausea
5%
Sinusitis
5%
Basal cell carcinoma
5%
Neuropathy peripheral
5%
Hyperuricaemia
3%
Paraesthesia
3%
Cystitis
3%
Bronchitis
3%
Blood creatine phosphokinase increased
3%
Skin ulcer
3%
Abdominal pain upper
3%
Pulmonary embolism
3%
Pneumonia
3%
Influenza
3%
Myalgia
3%
Urinary tract infection
3%
Depression
2%
Localised infection
2%
Intervertebral disc protrusion
2%
Urethral stenosis
2%
Night sweats
2%
Acute pulmonary oedema
2%
Vertigo
2%
Peripheral artery thrombosis
2%
Ureterolithiasis
2%
Pericardial effusion
2%
Acute myocardial infarction
2%
Syncope
2%
Gastrooesophageal reflux disease
2%
General physical health deterioration
2%
Atrial fibrillation
2%
Cardiac disorder
2%
Mitral valve incompetence
2%
Vertigo positional
2%
Retinal artery occlusion
2%
Visual acuity reduced
2%
Gastrointestinal haemorrhage
2%
Oesophageal varices haemorrhage
2%
Lower respiratory tract infection
2%
Pyelonephritis
2%
Respiratory tract infection
2%
Sepsis
2%
Tendon rupture
2%
Ulna fracture
2%
Weight decreased
2%
Decreased appetite
2%
Hyponatraemia
2%
Blast cell crisis
2%
Bone marrow tumour cell infiltration
2%
Lung adenocarcinoma
2%
Metastases to spine
2%
Myelofibrosis
2%
Prostatic adenoma
2%
Squamous cell carcinoma of skin
2%
Nephrolithiasis
2%
Gamma-glutamyltransferase increased
2%
Haematocrit increased
2%
Musculoskeletal pain
2%
Ischaemic stroke
2%
Diabetes mellitus
100%
80%
60%
40%
20%
0%
Study treatment Arm
All Crossover Patients
Best Available Therapy
Ruxolitinib
Trial Design
1Treatment groups
Experimental Treatment
Group I: RuxolitinibExperimental Treatment1 Intervention
Ruxolitinib at 5 mg twice a day (BID) for 1 week and then at 10 mg BID for 13-24 weeks and 1 week of 5 mg BID before stopping ruxolitinib.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ruxolitinib
2018
Completed Phase 3
~1140
Find a Location
Who is running the clinical trial?
National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,840 Previous Clinical Trials
47,852,123 Total Patients Enrolled
Cornelia D Cudrici, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- Your kidney function is very low, with an eGFR/CreatCr level less than 30 mL/min.I am a 58-year-old male with CNS Kohlmeier Degos Disease.Your platelet count is less than 50,000 per microliter of blood.Your white blood cell count is too low.Your hemoglobin level is less than 7 grams per deciliter.I do not have any life-threatening infections.You have a low level of lymphocytes in your blood.Your liver function test results are more than three times the normal limit.
Research Study Groups:
This trial has the following groups:- Group 1: Ruxolitinib
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Might I possess the qualifications to join this research project?
"The criteria for entry into this medical trial is that the patient has a diagnosis of Degos disease and is between 18 and 100 years old. There are spaces available for approximately 1 individual to take part in this research."
Answered by AI
Is this clinical experiment seeking participants above the age of forty?
"The age range for individuals hoping to join the trial is 18 to 100, as set by the inclusion criteria."
Answered by AI
How many participants are there in the current research program?
"Affirmative. Records on clinicaltrials.gov affirm that this research endeavour, first posted on August 15th 2023, is actively recruiting candidates. A single individual needs to be found from a solitary medical centre."
Answered by AI
Are there still opportunities to enroll in this research investigation?
"Affirmative. Information from clinicaltrials.gov attests that this experimental trial, initially shared on August 15th 2023, is continuing to search for participants. A single patient will be recruited and monitored at one medical site."
Answered by AI
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