G23.9
Conditions
Interventions
Group 1: Observation of clinical (clinical examination, UPDRS, UHDRS, clinical global, impression, qualitiy of life, electrophyisiological data etc.) and paraclinical data (routine analysis for safet
Sponsors
Sektion für Translationale Neurodegeneration "Albrecht Kossel"Klinik und Poliklinik für NeurologieUniversitätsmedizin Rostock
Eligibility
Sex/Gender
All
Age
18 Years to 99 Years
Inclusion criteria
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
Exclusion criteria: not applicable
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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. 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. | — |
Secondary
| Measure | Time frame |
|---|---|
| not applicable, oberservational study | — |
Countries
Germany
Contacts
Public ContactAndreas Hermann
Sektion für Translationale Neurodegeneration "Albrecht Kossel"Klinik und Poliklinik für NeurologieUniversitätsmedizin Rostock
Outcome results
None listed