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

Purpose

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	Intervention	Phase
Huntington's Disease Chorea	Drug: AFQ056 Drug: Placebo	Phase 2


Study Type:	Interventional
Study Design:	Allocation: Randomized Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:

Genetics Home Reference related topics: Huntington disease McLeod neuroacanthocytosis syndrome chorea-acanthocytosis

MedlinePlus related topics: Huntington's Disease

Genetic and Rare Diseases Information Center resources: Huntington Disease

U.S. FDA Resources

Further study details as provided by Novartis:

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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Potential effect of AFQ056 on the motor, cognitive, behavioral and functional assessments using UHDRS. [ Time Frame: Day 1 to day 46 ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]


Enrollment:	44
Study Start Date:	November 2009
Primary Completion Date:	August 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: AFQ056A	Drug: AFQ056
Placebo Comparator: Placebo	Drug: Placebo

Eligibility


Ages Eligible for Study:	30 Years to 85 Years (Adult, Senior)
Genders Eligible for Study:	Both
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

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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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
Study First Received:	November 19, 2009
Last Updated:	September 22, 2011
Health Authority:	Germany: Federal Institute for Drugs and Medical Devices United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Novartis:


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

ClinicalTrials.gov processed this record on September 28, 2016