What side effects are associated with this type of intervention?

Ruxolitinib, a Janus kinase (JAK) inhibitor, is being studied for the treatment of Kohlmeier-Degos Disease. Common side effects of JAK inhibitors like Ruxolitinib include anemia, thrombocytopenia, neutropenia, increased risk of infections, headache, dizziness, and elevated liver enzymes. Patients may also experience gastrointestinal symptoms such as diarrhea and nausea. It is important to monitor for these side effects and manage them appropriately under the guidance of a healthcare provider.

What prior FDA approvals exist?

There are no known FDA-approved treatments specifically for Kohlmeier-Degos Disease, particularly for cases affecting the central nervous system. The trial mentioned is investigating the use of Ruxolitinib, a Janus kinase (JAK) inhibitor, which is being tested for its efficacy in treating this rare condition. Other JAK inhibitors have not been mentioned in the context provided.

What other types of research exist?

Currently, there are no specific novel alternatives to Ruxolitinib mentioned for Kohlmeier Degos Disease in the provided context. Patients should consult their healthcare provider for the latest treatment options and ongoing clinical trials.

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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

Must not have

Active life-threatening infections

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months

Awards & highlights

No Placebo-Only Group

Summary

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 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.

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.

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.

Eligibility Criteria

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I do not have any life-threatening infections.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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 • NCT02038036

33%

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

Thrombocytosis

Upper respiratory tract infection

Hypercholesterolaemia

Haematoma

Dyslipidaemia

Pain in extremity

Abdominal discomfort

Diarrhoea

Dyspepsia

Vomiting

Blood lactate dehydrogenase increased

Memory impairment

Dyspnoea

Tinnitus

Osteoarthritis

Leukocytosis

Thrombocytopenia

Flatulence

Nausea

Sinusitis

Basal cell carcinoma

Neuropathy peripheral

Hyperuricaemia

Paraesthesia

Bronchitis

Cystitis

Blood creatine phosphokinase increased

Skin ulcer

Abdominal pain upper

Pulmonary embolism

Pneumonia

Influenza

Myalgia

Urinary tract infection

Depression

Acute pulmonary oedema

Peripheral artery thrombosis

Vertigo

Night sweats

Intervertebral disc protrusion

Urethral stenosis

Ureterolithiasis

Localised infection

Pericardial effusion

Acute myocardial infarction

Syncope

Gastrooesophageal reflux disease

General physical health deterioration

Atrial fibrillation

Cardiac disorder

Mitral valve incompetence

Vertigo positional

Retinal artery occlusion

Visual acuity reduced

Gastrointestinal haemorrhage

Oesophageal varices haemorrhage

Lower respiratory tract infection

Pyelonephritis

Respiratory tract infection

Sepsis

Tendon rupture

Ulna fracture

Weight decreased

Decreased appetite

Hyponatraemia

Blast cell crisis

Bone marrow tumour cell infiltration

Lung adenocarcinoma

Metastases to spine

Myelofibrosis

Prostatic adenoma

Squamous cell carcinoma of skin

Nephrolithiasis

Gamma-glutamyltransferase increased

Haematocrit increased

Musculoskeletal pain

Ischaemic stroke

Diabetes mellitus

100%

80%

60%

40%

20%

Study treatment Arm

All Crossover Patients

Best Available Therapy

Ruxolitinib

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

~1170

0 / 1Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Ruxolitinib, a Janus kinase (JAK) inhibitor, works by blocking the JAK-STAT signaling pathway, which is involved in the inflammatory response and immune regulation. This pathway is often overactive in various inflammatory and autoimmune conditions. By inhibiting this pathway, ruxolitinib can reduce inflammation and potentially prevent the blockage of small blood vessels, which is a hallmark of Kohlmeier Degos Disease. This mechanism is particularly important for patients with Kohlmeier Degos Disease as it addresses the underlying inflammatory processes that contribute to the disease's progression and severity.

Systematic review with meta-analysis: efficacy and safety of oral Janus kinase inhibitors for inflammatory bowel disease.[Severe MDA5 dermatomyositis associated with cancer and controlled by JAK inhibitor].New and emerging combination therapies for esophageal cancer.