Status:
COMPLETED
JAK/STAT Inhibition in CNS Kohlmeier-Degos Disease
Lead Sponsor:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Kohlmeier Degos Disease With Neurologic Involvement
Eligibility:
All Genders
18-100 years
Phase:
PHASE1
PHASE2
Brief Summary
Background: Kohlmeier-Degos (K-D) is a rare disease that leads to the blockage of small blood vessels in many organs; these can include the skin, eyes, stomach, lungs, heart, and the brain and spinal...
Detailed Description
Study Description: This single patient protocol will provide off-label treatment with a JAK/STAT ( Janus kinases/ Signal transducer and Activator of Transcription proteins) inhibitor to a patient wit...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- The study design was constructed to treat one subject, a 58 year old male with CNS Kohlmeier Degos Disease. Therefore, there are no specific inclusion criteria.
- EXCLUSION CRITERIA:
- Active life-threatening infections
- Hemoglobin \<7 g/dL
- Platelet counts \< 50 K /mcL
- Neutropenia (ANC \<0.5 x k/mcL)
- Lymphopenia (ALC \<0.2 x k/mcL)
- LFTs (liver function test) \> 3 x time upper limit
- eGFR/CreatCr \< 30 mL/min
Exclusion
Key Trial Info
Start Date :
September 28 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 28 2025
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT05998395
Start Date
September 28 2023
End Date
February 28 2025
Last Update
April 20 2025
Active Locations (1)
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1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892