Therapeutic Targeting of Lyn Kinase with Nilotinib to Treat Chorea-Acanthocytosis - an observational, non-interventional study

Recruiting

• Conditions: G23.9; Degenerative disease of basal ganglia, unspecified

• Registration Number: DRKS00024187
• Lead Sponsor: Sektion für Translationale Neurodegeneration Albrecht KosselKlinik und Poliklinik für NeurologieUniversitätsmedizin Rostock

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-01-25
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

Patients suffering from chorea-acanthocytosis (confirmed by chorein western blot and/or genetic testing), off-label therapy with Nilotinib

Exclusion Criteria

not applicable

Study & Design

• Study Type: observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
ot applicable, observational study. Participants receive interventions (Nilotinib as individual off label treatment) as part of routine medical care, and readouts were obtained to analyze the effect of the intervention.<br>This includes e.g. observation of clinical (clinical examination, UPDRS, UHDRS, global clinical impression, quality of life, electrophysiological data) and paraclinical data (routine analysis for safety reasons, target engagement in the blood, e.g. lyn kinase activity analysis) during off-label treatment with Nilotinib as part of routine medical care.

Secondary Outcome Measures

Name	Time	Method
not applicable, oberservational study