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

Primary Purpose

Huntington's Disease, Chorea

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
AFQ056
Placebo
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Huntington's Disease focused on measuring Huntington's disease, Chorea, Dyskinesia

Eligibility Criteria

30 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

AFQ056A

Placebo

Arm Description

Outcomes

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.

Secondary Outcome Measures

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

Full Information

First Posted
November 19, 2009
Last Updated
September 22, 2011
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT01019473
Brief Title
Efficacy, Safety and Tolerability of AFQ056 in Patients With Huntington's Disease in Reducing Chorea
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 Type
Interventional

2. Study Status

Record Verification Date
September 2011
Overall Recruitment Status
Terminated
Study Start Date
November 2009 (undefined)
Primary Completion Date
August 2011 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Huntington's Disease, Chorea
Keywords
Huntington's disease, Chorea, Dyskinesia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
44 (Actual)

8. Arms, Groups, and Interventions

Arm Title
AFQ056A
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
AFQ056
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
Potential effect of AFQ056 on the motor, cognitive, behavioral and functional assessments using UHDRS.
Time Frame
Day 1 to day 46
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
30 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigator Site
City
Berlin
Country
Germany
Facility Name
Novartis Investigator Site
City
Bochum
Country
Germany
Facility Name
Novartis Investigator Site
City
Munster
Country
Germany
Facility Name
Novartis Investigator Site
City
Taufkirchen
Country
Germany
Facility Name
Novartis Investigator Site
City
Tubingen
Country
Germany
Facility Name
Novartis Investigator Site
City
Ulm
Country
Germany
Facility Name
Novartis Investigator Site
City
Birmingham
Country
United Kingdom
Facility Name
Novartis Investigator Site
City
Cardiff
Country
United Kingdom
Facility Name
Novartis Investigator Site
City
Manchester
Country
United Kingdom
Facility Name
Novartis Investigator Site
City
Oxford
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
25689146
Citation
Reilmann R, Rouzade-Dominguez ML, Saft C, Sussmuth SD, Priller J, Rosser A, Rickards H, Schols 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.
Results Reference
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Efficacy, Safety and Tolerability of AFQ056 in Patients With Huntington's Disease in Reducing Chorea

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