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

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ClinicalTrials.gov Identifier: NCT01019473

Recruitment Status : Terminated

First Posted : November 25, 2009

Last Update Posted : September 23, 2011

Sponsor:

Information provided by (Responsible Party):

Novartis ( Novartis Pharmaceuticals )

Study Description

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


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

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: Huntington disease McLeod neuroacanthocytosis syndrome

MedlinePlus related topics: Huntington's Disease

Genetic and Rare Diseases Information Center resources: Huntington Disease

U.S. FDA Resources

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

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 :

Potential effect of AFQ056 on the motor, cognitive, behavioral and functional assessments using UHDRS. [ Time Frame: 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 [ Time Frame: Day 1 to day 46 ]

Eligibility Criteria

Information from the National Library of Medicine

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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

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

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 ClinicalTrials.gov identifier (NCT number): NCT01019473

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

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 Key Record Dates
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

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