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

Study Description

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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.

Condition or disease	Intervention/treatment	Phase
Huntington's Disease; Chorea	Drug: AFQ056; Drug: Placebo	Phase 2

Study Design

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	44 participants
Allocation:	Randomized
Intervention Model:	Parallel Assignment
Masking:	Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:	Treatment
Official Title:	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
Study Start Date :	November 2009
Actual Primary Completion Date :	August 2011

Arms and Interventions

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm	Intervention/treatment
Experimental: AFQ056A	Drug: AFQ056
Placebo Comparator: Placebo	Drug: Placebo

Outcome Measures

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :

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

Secondary Outcome Measures :

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

Eligibility Criteria

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Ages Eligible for Study:	30 Years to 85 Years (Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

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

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/exclusion criteria may apply

Contacts and Locations

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Locations

Germany
Novartis Investigator Site
Berlin, Germany
Novartis Investigator Site
Bochum, Germany
Novartis Investigator Site
Munster, Germany
Novartis Investigator Site
Taufkirchen, Germany
Novartis Investigator Site
Tubingen, Germany
Novartis Investigator Site
Ulm, Germany
United Kingdom
Novartis Investigator Site
Birmingham, United Kingdom
Novartis Investigator Site
Cardiff, United Kingdom
Novartis Investigator Site
Manchester, United Kingdom
Novartis Investigator Site
Oxford, United Kingdom

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators

Study Director:	Novartis Pharmaceuticals	Novartis Pharmaceuticals

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Reilmann R, Rouzade-Dominguez ML, Saft C, Süssmuth SD, Priller J, Rosser A, Rickards H, Schöls L, Pezous N, Gasparini F, Johns D, Landwehrmeyer GB, Gomez-Mancilla B. A randomized, placebo-controlled trial of AFQ056 for the treatment of chorea in Huntington's disease. Mov Disord. 2015 Mar;30(3):427-31. doi: 10.1002/mds.26174. Epub 2015 Feb 17.

Responsible Party:	Novartis Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT01019473 History of Changes
Other Study ID Numbers:	CAFQ056A2207; 2009-011743-39
First Posted:	November 25, 2009
Last Update Posted:	September 23, 2011
Last Verified:	September 2011

Keywords provided by Novartis ( Novartis Pharmaceuticals ):

Huntington's disease; Chorea; Dyskinesia

Additional relevant MeSH terms:

Huntington Disease; Chorea; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Dyskinesias; Movement Disorders	Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Cognition Disorders; Neurocognitive Disorders; Mental Disorders; Neurologic Manifestations; Signs and Symptoms