Friedreich's AtaxiaPhase 2/3NCT00803868
Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia
A Phase 2/3 study testing varenicline in people with Friedreich's Ataxia, run by University of South Florida at 2 sites in United States.
Trial summary
The purpose of this study is to determine if varenicline is effective in treating symptoms of Friedreich's ataxia. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Friedreich's Ataxia
- What is being tested?
- varenicline
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 28 participants
- Where
- 2 sitesUnited States
- Study dates
- May 2009to May 2010
- Phase
- Phase 2/3
- 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 FA diagnosed by confirmed by genetic testing.
- Age 18 years to 80 years.
- Women who are not pregnant or breast feeding, and who do not intend to become pregnant. Women of child-bearing potential must use a reliable method of contraception and must provide a negative urine pregnancy test at entry into the study.
- CBC, CMP, and diabetes lab results not indicative of clinically relevant abnormalities (results within the past 6 months prior to screening). These would include but are not limited to:
- Electrolytes (Chloride, Sodium, Potassium) within laboratory defined normal limits.
- Hemoglobin, white cell count, platelet count and fasting glucose within laboratory defined normal limits. Creatinine must be (≤1.5 mg/dl).
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- ALT (6-40 u/l) and AST (10-30 u/l) must be less than 2 times normal limit
- Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
- Patient permission (informed consent).
- Ambulatory status: Half of the enrolled patients must be able to ambulate with or without assistance; half of the enrolled patients must be non-ambulatory.
Who cannot join
- Any unstable illness that in the investigator's opinion preclude participation in this study.
- Use of another investigational product within the past 28 days.
- Patients with a history of substance abuse.
- Presence of preexisting psychiatric illness (specifically schizophrenia, bipolar disorder, or history of suicide attempt).
- Presence of diabetes (as determined by fasting blood glucose labs within the past 6 months).
- Presence of clinically significant cardiac disease (as determined by the investigator based on EKG and echocardiogram results within the past 6 months). Specifically, patients with an ejection fraction <40% or a prolonged QT interval (>50% of cycle duration) will be excluded. If abnormalities are noted on the EKG or echocardiogram, the patient will be eligible IF they provide clearance from a cardiologist.
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- Presence of current uncontrolled depression as measured by PHQ9 (criteria for depression include all of the following to be present: At least one of the first two questions on PHQ9 endorsed as positive (little pleasure, feeling depressed) indicating the symptom has been present more than half the time in the past two weeks; Question 10 about difficulty at work or home or getting along with others should be answered at least "somewhat difficult."; and the total score ≥ 10.
- Concurrent treatment with any MAOIs, Wellbutrin, or nicotine patches.
- Patients who currently smoke or have smoked within the past 12 months (Smoking will be defined as having smoked any substance even on a single occasion).
- Dementia or other psychiatric illness that prevents the patient from giving informed consent (MMSE less than 25).
- Legal incapacity or limited legal capacity.
- Presence of severe renal disease (creatinine >1.6) or hepatic disease (AST or ALT>2x times normal) (as evidenced by labs reported within the past 6 months).
- Abnormal WBC hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).
Trial locations and contacts
2 sites in 1 country.
United States2 sites
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of South Florida
Tampa, Florida, 33612
About this study
The purpose of this study is to determine if varenicline is effective in treating symptoms of Friedreich's ataxia.
- Conditions
- Friedreich's Ataxia
- Being tested
- varenicline, placebo
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vareniclineDrug
up to 1mg po bid for 9 weeks
placeboDrug
placebo matching study drug up to 1mg po bid
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Varenicline Receives: Drug: varenicline |
| 2 | Placebo comparator | Placebo Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Friedreich Ataxia Rating Scale (FARS)
Measured: 9 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2008 (estimated)
- Study startMay 2009
- Primary completionMay 2010
- Study completionMay 2010
Why it stopped: The DSMB recommended that the study be stopped as a result of concerns regarding safety and intolerability and insufficient evidence of efficacy.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich's AtaxiaStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Friedreich's Ataxia Research Alliance, Pfizer
- Responsible party
- Sponsor
- Investigators
- Theresa Zesiewicz, M.D., Principal investigator, University of South Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00803868
- Sponsor's protocol ID
- 6
- Status last verified
- Jul 2012
- First submitted
- 4 Dec 2008
- Last updated
- 24 Jul 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT00803868
- Acronym / sponsor ID
- 6
- Conditions
- Friedreich's Ataxia
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The DSMB recommended that the study be stopped as a result of concerns regarding safety and intolerability and insufficient evidence of efficacy.
- Enrolment
- 28 participants (actual)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- Collaborators
- Friedreich's Ataxia Research Alliance, Pfizer
- Study start
- May 2009
- Primary completion
- May 2010 (actual)
- Study completion
- May 2010 (actual)
- First posted
- 8 Dec 2008 (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.
The purpose of this study is to determine if varenicline is effective in treating symptoms of Friedreich's ataxia.
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 |
|---|---|---|
| 1 | Experimental | Drug: vareniclineVarenicline |
| 2 | Placebo comparator | Drug: placeboPlacebo |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Outpatients with FA diagnosed by confirmed by genetic testing.
- Age 18 years to 80 years.
- Women who are not pregnant or breast feeding, and who do not intend to become pregnant. Women of child-bearing potential must use a reliable method of contraception and must provide a negative urine pregnancy test at entry into the study.
- CBC, CMP, and diabetes lab results not indicative of clinically relevant abnormalities (results within the past 6 months prior to screening). These would include but are not limited to:
- Electrolytes (Chloride, Sodium, Potassium) within laboratory defined normal limits.
Key exclusion criteria (13 listed)
- Any unstable illness that in the investigator's opinion preclude participation in this study.
- Use of another investigational product within the past 28 days.
- Patients with a history of substance abuse.
- Presence of preexisting psychiatric illness (specifically schizophrenia, bipolar disorder, or history of suicide attempt).
- Presence of diabetes (as determined by fasting blood glucose labs within the past 6 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 po bid for 9 weeks | Drug | 1 |
| placeboplacebo matching study drug up to 1mg po bid | Drug | 2 |
Primary outcome
Friedreich Ataxia Rating Scale (FARS)
Time frame: 9 weeks
Results and safety
The study was stopped early (reason given: The DSMB recommended that the study be stopped as a result of concerns regarding safety and intolerability and insufficient evidence of efficacy.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 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, M.D. principal investigator
University of South Florida
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing varenicline in people with Friedreich's Ataxia, run by University of South Florida at 2 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 10 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: stopped early, as of the sponsor's last update on 24 Jul 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 (reason given: The DSMB recommended that the study be stopped as a result of concerns regarding safety and intolerability and insufficient evidence of efficacy.). 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?
Friedreich Ataxia Rating Scale (FARS) (time frame: 9 weeks). 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 24 Jul 2012.