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

• Register: EUCTR
• Last refreshed on: 12 November 2012
• Main ID: EUCTR2009-011743-39-DE
• Date of registration: 20/08/2009
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: Efficacy, Safety and Tolerability of AFQ056 in Patients with Huntington's Disease in Reducing Chorea
• Scientific 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
• Date of first enrolment: 18/09/2009
• Target sample size: 60
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-011743-39
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Germany, United Kingdom, United States

Contacts

• Name: Medizinischer Infoservice
• Affiliation: Novartis Pharma GmbH

• Name: Medizinischer Infoservice
• Affiliation: Novartis Pharma GmbH

Key inclusion & exclusion 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
Other protocol-defined inclusion criteria may apply


Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 60
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

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 exclusion criteria may apply



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Huntington's disease
MedDRA version: 13.1 Level: LLT Classification code 10020469 Term: Huntington's chorea System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: AFQ056
Product Code: AFQ056
Pharmaceutical Form: Capsule, hard
CAS Number: 543906-09-8
Current Sponsor code: AFQ056
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 25-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Product Name: AFQ056
Product Code: AFQ056
Pharmaceutical Form: Capsule, hard
CAS Number: 543906-09-8
Current Sponsor code: AFQ056
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: To assess the efficacy of AFQ056 on the severity of chorea in Huntington’s disease patients in clinical stages I-III on day 28, using the Unified Huntington’s Disease Rating Scale (UHDRS) Maximal Chorea score and * the orientation index from the quantitative grip force motor assessment.

*Both co-primary objectives will be assessed but achivement of one OR the other co-primary endpoint is sufficient to achive PoC.

To assess the safety and tolerability of multiple titrated doses of AFQ056 in Huntington’s disease patients in clinical stages I-III.

Primary end point(s): 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 Objective: - To assess the potential effect of multiple titrated doses of AFQ056 on the motor, cognitive, behavioral and functional assessments using the Unified Huntington’s Disease Rating Scale (UHDRS).

- To assess the potential effect of multiple titrated doses of AFQ056 on Functional and Quality of Life scales, neuropsychiatric assessments and cognitive assessments in Huntington’s Disease patients in clinical stages I-III

Timepoint(s) of evaluation of this end point: Baseline to day 28

Secondary Outcome(s)

Secondary end point(s): - Safety and tolerability of AFQ056 in Huntington's disease patients

- 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

Timepoint(s) of evaluation of this end point: - Safety and tolerability: Day 1 to day 46

- Potential Effect on the mmotor, cognitive, behavioral and functional assessments using UHDRS: 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 : Day 1 to day 46

Secondary ID(s)

NCT01019473

CAFQ056A2207

Source(s) of Monetary Support

Novartis Pharma Services AG