Clinical trial NCT01019473: 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
Overall status: TERMINATED
Phase: PHASE2
Conditions: Huntington's Disease
Chorea
Keywords: Huntington's disease
Chorea
Dyskinesia
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2009-11-25
Enrollment: 44
Sex: ALL
Minimum age (years): 30
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Efficacy of AFQ056 on the severity of chorea in Huntington's disease measured by Unified Huntington's Disease Rating Scale (UHDRS) Maximal Chorea score.
timeFrame: Baseline to day 28
Secondary outcomes:
measure: Potential effect of AFQ056 on the motor, cognitive, behavioral and functional assessments using UHDRS.
timeFrame: Day 1 to day 46
measure: Potential effect of AFQ056 on functional and quality of life scales, neuropsychiatric assessments and cognitive assessments in Huntington's Disease patients
timeFrame: Day 1 to day 46
Other outcomes:
Locations:
city: Berlin
country: Germany
facility: Novartis Investigator Site
city: Bochum
country: Germany
facility: Novartis Investigator Site
city: Munster
country: Germany
facility: Novartis Investigator Site
city: Taufkirchen
country: Germany
facility: Novartis Investigator Site
city: Tubingen
country: Germany
facility: Novartis Investigator Site
city: Ulm
country: Germany
facility: Novartis Investigator Site
city: Birmingham
country: United Kingdom
facility: Novartis Investigator Site
city: Cardiff
country: United Kingdom
facility: Novartis Investigator Site
city: Manchester
country: United Kingdom
facility: Novartis Investigator Site
city: Oxford
country: United Kingdom
facility: Novartis Investigator Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
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.