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NCT05998395 · ClinicalTrials.gov registry record · Phase 1
JAK/STAT Inhibition in CNS Kohlmeier-Degos Disease
A Phase 1 study of Kohlmeier-Degos Disease and Kohlmeier Degos Disease With Neurologic Involvement, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
- 1
- Study location
NCT05998395: Completed Phase 1 study of Kohlmeier-Degos Disease and Kohlmeier Degos Disease With Neurologic Involvement, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT05998395 is a Phase 1 study of Kohlmeier-Degos Disease and Kohlmeier Degos Disease With Neurologic Involvement that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 1 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (98% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05998395, a Phase 1 study of Kohlmeier-Degos Disease and Kohlmeier Degos Disease With Neurologic Involvement, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 1 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: Kohlmeier-Degos (K-D) is a rare disease that leads to the inflammation and/or blockage of small blood vessels in many organs; these can include the skin, eyes (rare), small bowels, lungs, heart, and the brain and spinal cord (central nervous system, or CNS). There are no known effective treatments for K-D that affects the CNS. Objective: To test a drug (ruxolitinib) in a person with K-D affecting the CNS. Eligibility: This study is designed to treat 1 adult participant with K-D affecting the CNS. Design: The participant will be screened: They will have a physical exam and blood tests. They will have skin biopsies: Small samples of skin will be removed. They will have a lumbar puncture: A needle will be inserted in his back to draw fluid from the space around the spinal cord. They will have a magnetic resonance imaging (MRI) scan: they will lie on a table that slides into a tube to take pictures of their brain and spinal cord. They will see a doctor who specializes in nerves. 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 have up to 7 clinic visits in 28 weeks. Each visit will be 1 to 3 days. MRI scans, biopsies, lumbar punctures, and other blood tests will be repeated on different visits. The participant may receive follow-up phone calls between visits. The participant will report any adverse effects. Unscheduled visits may be needed if new symptoms develop. The last follow-up will be 4 weeks after the last dose of the study drug.
Primary Outcome
The primary endpoint is stability or regression of existing enhancing lesions or no development of new enhancing lesions in the brain and spine observed in MRI evaluation after 13 weeks and up to 73 weeks of ruloxitinib (10 mg BID) compared to pre-treatment MRI images.
Conditions Studied
Interventions
- DRUG Ruxolitinib
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2023-09-28 |
| Est. Completion | 2025-02-28 |
| Phase | Phase 1 |
What the finished NCT05998395 record still lists
NCT05998395 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 1 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (98% lower).
The record links to 2 conditions, with Kohlmeier-Degos Disease appearing as the primary indexed condition, and to 1 intervention - of which Ruxolitinib is the first listed.
NCT05998395 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT05998395 about?
NCT05998395 is a clinical study titled "JAK/STAT Inhibition in CNS Kohlmeier-Degos Disease". Background: Kohlmeier-Degos (K-D) is a rare disease that leads to the inflammation and/or blockage of small blood vessels in many organs; these can include the skin, eyes (rare), small bowels, lungs, heart, and the brain and spinal cord (central nervous system, or CNS). There are no known effective...
What is the current status of trial NCT05998395?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2023-09-28. Estimated completion is 2025-02-28.
What conditions does trial NCT05998395 study?
This clinical trial studies the following conditions: Kohlmeier-Degos Disease, Kohlmeier Degos Disease With Neurologic Involvement.
What interventions are being tested in trial NCT05998395?
The interventions under investigation include: Ruxolitinib (DRUG).
Who is sponsoring clinical trial NCT05998395?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05998395 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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