Using baricitinib to treat Kohlmeier-Degos disease with neurological symptoms
A Phase IIa Trial of Baricitinib in the Treatment of Kohlmeier-Degos Disease Patients With Neurological Involvement
PHASE2 · National Institutes of Health Clinical Center (CC) · NCT06923072
This study is testing if the medication baricitinib can help adults with Kohlmeier-Degos disease who have neurological symptoms feel better over 24 weeks.
Quick facts
| Phase | PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 18 Years to 99 Years |
| Sex | All |
| Sponsor | National Institutes of Health Clinical Center (CC) (nih) |
| Drugs / interventions | baricitinib |
| Locations | 1 site (Bethesda, Maryland) |
| Trial ID | NCT06923072 on ClinicalTrials.gov |
What this trial studies
This phase II clinical trial aims to evaluate the efficacy of baricitinib, an FDA-approved medication, in patients suffering from Kohlmeier-Degos disease (KD) who exhibit neurological involvement. Participants aged 18 and older will undergo a series of evaluations, including imaging and lumbar punctures, before receiving baricitinib for 24 weeks, alongside their usual medications. The study will assess the progression of neurological symptoms and patient-reported outcomes over a total of 40 weeks. The hypothesis is that baricitinib will reduce disease progression by targeting specific immune signaling pathways.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older diagnosed with Kohlmeier-Degos disease who have neurological abnormalities.
Not a fit: Patients with active infections or those unable to comply with the study procedures may not benefit from this trial.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the management of neurological symptoms in patients with Kohlmeier-Degos disease.
How similar studies have performed: While baricitinib has been effective in other conditions, this specific application for Kohlmeier-Degos disease is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
* INCLUSION CRITERIA: In order to be eligible to participate in this study, an individual must meet all of the following criteria: * Provision of signed and dated informed consent form by the subject or Legally Authorized Representative (LAR). * Stated willingness to comply with all study procedures and availability for the duration of the study. * Male or female, aged 18 or older. * Subjects diagnosed with systemic Degos disease, who manifest neurologic abnormalities observed clinically, radiologically or in abnormal laboratory findings. * Ability to take oral medication and be willing to adhere to the baricitinib regimen. * For female patients of reproductive potential, non-pregnant, non-breastfeeding: agree to use of highly effective contraception for the duration of the study and 30 days after the last dose. * Ability of subject or LAR to understand and the willingness to sign a written informed consent document. EXCLUSION CRITERIA: * Active infection not responding to appropriate therapy * Hemoglobin \<7 g/dL * Platelet counts \< 50 K /mcL * Neutropenia (ANC \<0.5 x k/mcL) * Lymphopenia (Absolute Lymphocyte Count \[ALC\] \<0.2x k/mcL) * Liver function tests (LFTs \> 2x time upper limit of normal) * Estimated Glomerular Filtration Rate (eGFR)/Creatinine (Cr \< 30 mL/min) * Have experienced any of the following within 12 weeks of screening: VTE (DVT/pulmonary embolism \[PE\]), myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage III/IV heart failure. * Have had symptomatic herpes zoster infection within 12 weeks prior to a enrolling in the study . * Have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB. * Have evidence of active TB or latent TB * Have been exposed to a live vaccine within 12 weeks of baricitinib treatment or are expected to need/receive a live vaccine during the course of the study * No gadolinium based contrast agent exposure is permitted if eGFR \< 30 mL/min/1.73m\^2 using the CKD-EPI equation measured within 5 days . * Breast feeding * Pregnancy * Uncontrolled malignancy
Where this trial is running
Bethesda, Maryland
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (RECRUITING)
Study contacts
- Principal investigator: Cornelia D Cudrici, M.D. — National Heart, Lung, and Blood Institute (NHLBI)
- Study coordinator: William R Whalen, C.R.N.P.
- Email: william.whalen@nih.gov
- Phone: (301) 402-9841
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Kohlmeier-Degos Disease, Malignant Atrophic Papulosis, Degos Disease, Papulosis, Malignant Atrophic, Kohlmeier-Degos, Baricitinib, Neurological involvement of Degos Disease