Spinocerebellar Ataxia Type 3Phase 2NCT00992771
Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3
A Phase 2 study testing varenicline in people with Spinocerebellar Ataxia Type 3, run by University of South Florida at 3 sites in United States.
Trial summary
Spinocerebellar ataxia (SCA) is a group of inherited disorders characterized by cerebellar degeneration leading to imbalance, incoordination, speech difficulties and problems with walking. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Spinocerebellar Ataxia Type 3
- What is being tested?
- varenicline
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 3 sitesUnited States
- Study dates
- Oct 2009to Apr 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Outpatients with spinocerebellar ataxia type 3 diagnosed by a movement disorder specialist and confirmed by genetic testing (of the patient or in a first degree relative of the patient).
- Age 18 years to 80 years.
- Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
- Serum creatine kinase, complete metabolic panel, complete blood count, liver function tests, renal function tests, platelets and EKG are within normal limits (results obtained from primary care physician and dated within the past 6 months or obtained at screening visit).
- Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
- Ability to ambulate with or without assistance.
- Score of 10 or higher (worse) on the SARA total score.
- Score of 3 or higher (worse) on the 'gait' subsection of the SARA rating scale.
Who cannot join
- Any unstable illness or concomitant medical condition that, in the investigator's opinion, precludes participation in this study. This includes other disorders that may affect gait or balance (stroke, arthritis, etc).
- Pregnancy or lactation.
- Concurrent participation in another clinical study.
- Patients with a history of substance abuse.
- Patients who currently smoke or have smoked within the past 12 months.
- Presence of psychosis, bipolar disorder, untreated depression (BDI greater than or equal to 21), or history of suicide attempt.
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- Concurrent treatment with any MAOIs, Wellbutrin, or nicotine patches.
- Dementia or other psychiatric illness that prevents the patient from giving informed consent (Mini Mental Status Exam score less than 24).
- Legal incapacity or limited legal capacity.
- Presence of severe renal disease (BUN 50% greater than normal or creatinine clearance <60 mL/min) or hepatic disease.
- Abnormal creatine kinase and/or platelet count in the past 6 months (as determined by lab reports obtained from primary care physicians or conducted at baseline).
- Use of varenicline within the previous 30 days.
- Ataxia derived from any other cause than genetically-confirmed SCA (including but not limited to alcoholism, head injury, Multiple Sclerosis, olivo-ponto-cerebellar atrophy or multiple system atrophy).
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of Florida
Gainesville, Florida, 32610
University of California - Los Angeles
Los Angeles, California, 90095
University of South Florida
Tampa, Florida, 33612
About this study
Official title: A Pilot, Randomized, Double-blind, Placebo-controlled Phase I Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3
Spinocerebellar ataxia (SCA) is a group of inherited disorders characterized by cerebellar degeneration leading to imbalance, incoordination, speech difficulties and problems with walking. Recently, individual case reports have suggested that varenicline, a drug used in smoking cessation, produces substantial improvement in patients with several inherited ataxias. A modest response was noted in 5 patients with SCA, suggesting that it is potentially efficacious in this disorder as well. Although this agent is available for off-label use, the severe side effects noted with its use and the lack of long-term toxicity data demand that it be systematically assessed. The present study will test whether varenicline is safe and potentially efficacious in a heterogeneous cohort of adults with SCA.
- Conditions
- Spinocerebellar Ataxia Type 3
- Being tested
- varenicline, placebo
- Medical subject headings
- Machado-Joseph Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vareniclineDrug
up to 1mg BID for 8 weeks
placeboDrug
placebo matching varenicline, up to 1mg BID for 8 weeks
| Study group | Role | What they receive |
|---|---|---|
| Varneicline | Experimental | Not described Receives: Drug: varenicline |
| 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.
Changes in the patient's SARA Rating Scale total score
Measured: 25 weeks
Frequency and severity of dose-limiting adverse events
Measured: 25 weeks
Secondary outcomes (3)
The effect of varenicline on quality of life in patients with spinocerebellar ataxia
Measured: 25 weeks
The effect of varenicline on depression and anxiety ratings
Measured: 25 weeks
The effect of varenicline on the activity of daily living (ADL) in patients with spinocerebellar ataxia
Measured: 25 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2009 (estimated)
- Study startOct 2009
- Primary completionApr 2011
- Study completionApr 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinocerebellar Ataxia Type 3Studies of the same condition, open and under way.
- More trials run by University of South FloridaThe 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
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- Collaborators
- National Ataxia Foundation, Bob Allison Ataxia Research Center (BAARC), Pfizer
- Responsible party
- Principal investigator, Theresa Zesiewicz, University of South Florida
- Investigators
- Theresa Zesiewicz, MD, Principal investigator, University of South Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00992771
- Sponsor's protocol ID
- 8
- Status last verified
- Jun 2012
- First submitted
- 8 Oct 2009
- Last updated
- 18 Jun 2012
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot, Randomized, Double-blind, Placebo-controlled Phase I Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3
- ClinicalTrials.gov ID
- NCT00992771
- Acronym / sponsor ID
- 8
- Conditions
- Spinocerebellar Ataxia Type 3
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- Collaborators
- National Ataxia Foundation, Bob Allison Ataxia Research Center (BAARC), Pfizer
- Study start
- Oct 2009
- Primary completion
- Apr 2011 (actual)
- Study completion
- Apr 2011 (actual)
- First posted
- 9 Oct 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.
Spinocerebellar ataxia (SCA) is a group of inherited disorders characterized by cerebellar degeneration leading to imbalance, incoordination, speech difficulties and problems with walking. Recently, individual case reports have suggested that varenicline, a drug used in smoking cessation, produces substantial improvement in patients with several inherited ataxias. A modest response was noted in 5 patients with SCA, suggesting that it is potentially efficacious in this disorder as well. Although this agent is available for off-label use, the severe side effects noted with its use and the lack of long-term toxicity data demand that it be systematically assessed. The present study will test whether varenicline is safe and potentially efficacious in a heterogeneous cohort of adults with SCA.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Varneicline | Experimental | Drug: varenicline |
| Placebo | Placebo comparator | Drug: placebo |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Outpatients with spinocerebellar ataxia type 3 diagnosed by a movement disorder specialist and confirmed by genetic testing (of the patient or in a first degree relative of the patient).
- Age 18 years to 80 years.
- Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
- Serum creatine kinase, complete metabolic panel, complete blood count, liver function tests, renal function tests, platelets and EKG are within normal limits (results obtained from primary care physician and dated within the past 6 months or obtained at screening visit).
- Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
Key exclusion criteria (13 listed)
- Any unstable illness or concomitant medical condition that, in the investigator's opinion, precludes participation in this study. This includes other disorders that may affect gait or balance (stroke, arthritis, etc).
- Pregnancy or lactation.
- Concurrent participation in another clinical study.
- Patients with a history of substance abuse.
- Patients who currently smoke or have smoked within the past 12 months.
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 |
|---|---|---|
| vareniclineup to 1mg BID for 8 weeks | Drug | Varneicline |
| placeboplacebo matching varenicline, up to 1mg BID for 8 weeks | Drug | Placebo |
Primary outcome
Changes in the patient's SARA Rating Scale total score
Time frame: 25 weeks
Frequency and severity of dose-limiting adverse events
Time frame: 25 weeks
3 secondary outcomes
The effect of varenicline on quality of life in patients with spinocerebellar ataxia
Time frame: 25 weeks
The effect of varenicline on depression and anxiety ratings
Time frame: 25 weeks
The effect of varenicline on the activity of daily living (ADL) in patients with spinocerebellar ataxia
Time frame: 25 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2012.
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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.
Theresa Zesiewicz, MD principal investigator
University of South Florida
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing varenicline in people with Spinocerebellar Ataxia Type 3, run by University of South Florida at 3 sites in United States.
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 8 inclusion and 13 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: finished, as of the sponsor's last update on 18 Jun 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Changes in the patient's SARA Rating Scale total score (time frame: 25 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 18 Jun 2012.