Therapeutic Targeting of Lyn Kinase To Treat Chorea-Acanthocytosis - an observational, non-interventional study

Recruiting

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

• Registration Number: DRKS00023177
• 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: 2020-10-05
• 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 testin), off-label therapy with Dasatinib or Nilotinib

Exclusion Criteria

not applicable

Study & Design

• Study Type: observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
not applicable, oberservational study, participants receive/received interventions (Dasatinib/Nilotinib as individual off label treatment) as part of routine medical care, and readouts were sampled to analyse the effect of the intervention. <br>This includes e.g. oberservation of clinical (clinical examination, UPDRS, UHDRS, global clinical impression, qualitiy of life, electrophysiological data) and paraclinical data (routine analysis for safety reasons, target engagement in the blood, e.g. lyn kinase activity analyisis) under off-label treatment with Dasatinib or Nilotinib.<br>

Secondary Outcome Measures

Name	Time	Method
not applicable, oberservational study