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

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2009-011743-39-DE

Enrollment

Registered

2009-08-20

Start date

2009-09-18

Completion date

Unknown

Last updated

2012-11-12

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

Conditions

Huntington's disease MedDRA version: 13.1 Level: LLT Classification code 10020469 Term: Huntington's chorea System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: AFQ056 Product Code: AFQ056 Pharmaceutical Form: Capsule, hard CAS Number: 543906-09-8 Current Sponsor code: AFQ056 Concentration unit: mg milligram(s) Concentration type: equal Concentr

Eligibility

Sex/Gender

All

Inclusion criteria

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 Other protocol-defined inclusion criteria may apply Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 60 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range

Exclusion criteria

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 exclusion criteria may apply

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the efficacy of AFQ056 on the severity of chorea in Huntington’s disease patients in clinical stages I-III on day 28, using the Unified Huntington’s Disease Rating Scale (UHDRS) Maximal Chorea score and * the orientation index from the quantitative grip force motor assessment. *Both co-primary objectives will be assessed but achivement of one OR the other co-primary endpoint is sufficient to achive PoC. To assess the safety and tolerability of multiple titrated doses of AFQ056 in Huntington’s disease patients in clinical stages I-III. ;Secondary Objective: - To assess the potential effect of multiple titrated doses of AFQ056 on the motor, cognitive, behavioral and functional assessments using the Unified Huntington’s Disease Rating Scale (UHDRS). - To assess the potential effect of multiple titrated doses of AFQ056 on Functional and Quality of Life scales, neuropsychiatric assessments and cognitive assessments in Huntington’s Disease patients in clinical stages I-III ;Primary end point(s): Efficacy of AFQ056 on the severity of chorea in Huntington's disease measured by Unified Huntington's Disease Rating Scale (UHDRS) Maximal Chorea score. ;Timepoint(s) of evaluation of this end point: Baseline to day 28	—

Measure

Time frame

Main Objective: To assess the efficacy of AFQ056 on the severity of chorea in Huntington’s disease patients in clinical stages I-III on day 28, using the Unified Huntington’s Disease Rating Scale (UHDRS) Maximal Chorea score and * the orientation index from the quantitative grip force motor assessment. *Both co-primary objectives will be assessed but achivement of one OR the other co-primary endpoint is sufficient to achive PoC. To assess the safety and tolerability of multiple titrated doses of AFQ056 in Huntington’s disease patients in clinical stages I-III. ;Secondary Objective: - To assess the potential effect of multiple titrated doses of AFQ056 on the motor, cognitive, behavioral and functional assessments using the Unified Huntington’s Disease Rating Scale (UHDRS). - To assess the potential effect of multiple titrated doses of AFQ056 on Functional and Quality of Life scales, neuropsychiatric assessments and cognitive assessments in Huntington’s Disease patients in clinical stages I-III ;Primary end point(s): Efficacy of AFQ056 on the severity of chorea in Huntington's disease measured by Unified Huntington's Disease Rating Scale (UHDRS) Maximal Chorea score. ;Timepoint(s) of evaluation of this end point: Baseline to day 28

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Secondary

Measure	Time frame
Secondary end point(s): - Safety and tolerability of AFQ056 in Huntington's disease patients - Potential effect of AFQ056 on the motor, cognitive, behavioral and functional assessments using UHDRS. - Potential effect of AFQ056 on functional and quality of life scales, neuropsychiatric assessments and cognitive assessments in Huntington's Disease patients ;Timepoint(s) of evaluation of this end point: - Safety and tolerability: Day 1 to day 46 - Potential Effect on the mmotor, 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	—

Measure

Time frame

Secondary end point(s): - Safety and tolerability of AFQ056 in Huntington's disease patients - Potential effect of AFQ056 on the motor, cognitive, behavioral and functional assessments using UHDRS. - Potential effect of AFQ056 on functional and quality of life scales, neuropsychiatric assessments and cognitive assessments in Huntington's Disease patients ;Timepoint(s) of evaluation of this end point: - Safety and tolerability: Day 1 to day 46 - Potential Effect on the mmotor, 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

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Countries

Germany, United Kingdom, United States

Contacts

Public ContactMedizinischer Infoservice

Novartis Pharma GmbH

infoservice.novartis@novartis.com+49 01802 232300

Outcome results

None listed

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