Huntington's DiseasePhase 2NCT01019473
Efficacy, Safety and Tolerability of AFQ056 in Patients With Huntington's Disease in Reducing Chorea
A Phase 2 study testing AFQ056 in people with Huntington's Disease and Chorea, run by Novartis Pharmaceuticals at 10 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 30 to 85 years
- Conditions the study is for
- Huntington's Disease, Chorea
- What is being tested?
- AFQ056
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 44 participants
- Where
- 10 sitesin 2 countries
- Study dates
- Nov 2009to Aug 2011
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
Germany6 sites
Novartis Investigator Site
Berlin
Novartis Investigator Site
Bochum
Novartis Investigator Site
Münster
Novartis Investigator Site
Taufkirchen
Novartis Investigator Site
Tübingen
Novartis Investigator Site
Ulm
United Kingdom4 sites
Novartis Investigator Site
Birmingham
Novartis Investigator Site
Cardiff
Novartis Investigator Site
Manchester
Novartis Investigator Site
Oxford
About this study
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
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.
- Conditions
- Huntington's Disease, Chorea
- Being tested
- AFQ056, Placebo
- Keywords
- Huntington's disease, Chorea, Dyskinesia
- Medical subject headings
- Huntington Disease, Chorea, Dyskinesias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AFQ056Drug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| AFQ056A | Experimental | Not described Receives: Drug: AFQ056 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of AFQ056 on the severity of chorea in Huntington's disease measured by Unified Huntington's Disease Rating Scale (UHDRS) Maximal Chorea score.
Measured: Baseline to day 28
Secondary outcomes (2)
Potential effect of AFQ056 on the motor, cognitive, behavioral and functional assessments using UHDRS.
Measured: 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
Measured: Day 1 to day 46
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2009 (estimated)
- Study startNov 2009
- Primary completionAug 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Huntington's DiseaseStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01019473
- Sponsor's protocol ID
- CAFQ056A2207
- Other IDs
- 2009-011743-39, 2009
- Status last verified
- Sep 2011
- First submitted
- 19 Nov 2009
- Last updated
- 23 Sep 2011
- Results posted
- No
Publications
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.
PubMed 25689146
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01019473
- Acronym / sponsor ID
- CAFQ056A2207
- Conditions
- Huntington's Disease, Chorea
- Keywords
- Huntington's disease, Chorea, Dyskinesia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Nov 2009
- Primary completion
- Aug 2011 (actual)
- First posted
- 25 Nov 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AFQ056A | Experimental | Drug: AFQ056 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 30 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AFQ056 | Drug | AFQ056A |
| Placebo | Drug | Placebo |
Primary outcome
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
2 secondary outcomes
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
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2011.
- Derived from this study: PubMed 25689146Reilmann 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AFQ056 in people with Huntington's Disease and Chorea, run by Novartis Pharmaceuticals at 10 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 23 Sep 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Germany, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome 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. (time frame: Baseline to day 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01019473, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Sep 2011.