Efficacy, Safety and Tolerability of AFQ056 in Patients With Huntington's Disease in Reducing Chorea

A Multi-centre, Randomized, Double-blind, Placebo-controlled, Parallel-group, Multiple Oral Dose Titration Proof of Concept Study in Patients With Huntington's Disease to Assess the Efficacy, Safety and Tolerability of AFQ056 in Reducing Chorea

Status

Terminated

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01019473

Enrollment

Registered

2009-11-25

Start date

2009-11-30

Completion date

Unknown

Last updated

2011-09-23

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

Conditions

Chorea, Huntington's Disease

Keywords

Huntington's disease, Chorea, Dyskinesia

Brief summary

This study will assess the efficacy, safety and tolerability of AFQ056 when added to optimize standard therapy in patients that have Huntington's disease in reducing chorea.

Interventions

DRUGPlacebo

DRUGAFQ056

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

30 Years to 85 Years

Healthy volunteers

Inclusion criteria

* Huntington's disease (based on DNA testing polyQ \>36) with a UHDRS maximal chorea score of \>10 * patient with concomitant Huntington's medication (anti-depressants, neuroleptics, benzodiazepines) are allowed but the total daily dose and dosing regimen has to be stable for at least one months prior to randomization * female patients without childbearing potential (post-menopausal or surgically sterilized), all patients must using a double-barrier local contraception

Exclusion criteria

* patients with marked cognitive impairment (MMSE less than 18), with presence of psychosis and/or confusional states * patients with a history or presence of renal impairment and/or liver disease Other protocol-defined inclusion/

Design outcomes

Primary

Measure	Time frame
Efficacy of AFQ056 on the severity of chorea in Huntington's disease measured by Unified Huntington's Disease Rating Scale (UHDRS) Maximal Chorea score.	Baseline to day 28

Secondary

Measure	Time frame
Potential effect of AFQ056 on the motor, cognitive, behavioral and functional assessments using UHDRS.	Day 1 to day 46
Potential effect of AFQ056 on functional and quality of life scales, neuropsychiatric assessments and cognitive assessments in Huntington's Disease patients	Day 1 to day 46

Countries

Germany, United Kingdom

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026