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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01019473
Recruitment Status : Terminated
First Posted : November 25, 2009
Last Update Posted : September 23, 2011
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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

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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

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

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 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01019473

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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
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Novartis Pharmaceuticals Identifier: NCT01019473    
Other Study ID Numbers: CAFQ056A2207
First Posted: November 25, 2009    Key Record Dates
Last Update Posted: September 23, 2011
Last Verified: September 2011
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Huntington's disease
Additional relevant MeSH terms:
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Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders
Neurologic Manifestations