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

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

DRKS

Registry ID

DRKS00024187

Enrollment

Registered

2021-01-25

Start date

2021-02-01

Completion date

Unknown

Last updated

2025-04-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

G23.9

Interventions

Group 1: Observation of clinical (clinical examination, UPDRS, UHDRS, clinical global impression, quality of life, electrophysiological data, etc.) and paraclinical data (routine analysis for safety r

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 testing), off-label therapy with Nilotinib

Exclusion criteria

Exclusion criteria: not applicable

Design outcomes

Primary

Measure	Time frame
Not 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. 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.	—

Measure

Time frame

Not 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. 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

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

andreas.hermann@uni.med-rostock.de0049 381 494 9541

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Feb 4, 2026