Spinocerebellar Ataxia Type 3 Clinical Trial
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
Verified date | June 2012 |
Source | University of South Florida |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 20 |
Est. completion date | April 2011 |
Est. primary completion date | April 2011 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 80 Years |
Eligibility |
Inclusion Criteria: 1. 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). 2. Age 18 years to 80 years. 3. Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study. 4. 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). 5. Stable doses of all medications for 30 days prior to study entry and for the duration of the study. 6. Ability to ambulate with or without assistance. 7. Score of 10 or higher (worse) on the SARA total score. 8. Score of 3 or higher (worse) on the 'gait' subsection of the SARA rating scale. Exclusion Criteria: 1. 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). 2. Pregnancy or lactation. 3. Concurrent participation in another clinical study. 4. Patients with a history of substance abuse. 5. Patients who currently smoke or have smoked within the past 12 months. 6. Presence of psychosis, bipolar disorder, untreated depression (BDI greater than or equal to 21), or history of suicide attempt. 7. Concurrent treatment with any MAOIs, Wellbutrin, or nicotine patches. 8. Dementia or other psychiatric illness that prevents the patient from giving informed consent (Mini Mental Status Exam score less than 24). 9. Legal incapacity or limited legal capacity. 10. Presence of severe renal disease (BUN 50% greater than normal or creatinine clearance <60 mL/min) or hepatic disease. 11. 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). 12. Use of varenicline within the previous 30 days. 13. 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). |
Country | Name | City | State |
---|---|---|---|
United States | University of Florida | Gainesville | Florida |
United States | University of California - Los Angeles | Los Angeles | California |
United States | University of South Florida | Tampa | Florida |
Lead Sponsor | Collaborator |
---|---|
University of South Florida | Bob Allison Ataxia Research Center (BAARC), National Ataxia Foundation, Pfizer |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Changes in the patient's SARA Rating Scale total score | 25 weeks | ||
Primary | Frequency and severity of dose-limiting adverse events | 25 weeks | ||
Secondary | The effect of varenicline on quality of life in patients with spinocerebellar ataxia | 25 weeks | ||
Secondary | The effect of varenicline on depression and anxiety ratings | 25 weeks | ||
Secondary | The effect of varenicline on the activity of daily living (ADL) in patients with spinocerebellar ataxia | 25 weeks |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT01096082 -
Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3
|
Phase 2/Phase 3 | |
Active, not recruiting |
NCT03701399 -
Troriluzole in Adult Subjects With Spinocerebellar Ataxia
|
Phase 3 | |
Active, not recruiting |
NCT04229823 -
Natural History of Oculomotor Neurophysiology in Ataxic and Pre-ataxic Carriers of SCA3/MJD
|
||
Active, not recruiting |
NCT04419974 -
Astrocytic Markers and the Pre-ataxic Period of SCA3/MJD - BIGPRO Study Astrocytes
|
||
Terminated |
NCT05490563 -
STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia
|
Phase 2/Phase 3 | |
Withdrawn |
NCT04301284 -
Study of CAD-1883 for Spinocerebellar Ataxia
|
Phase 2 | |
Withdrawn |
NCT01096095 -
Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
|
Phase 2 | |
Active, not recruiting |
NCT04268147 -
Instrumented Data Exchange for Ataxia Study
|
||
Recruiting |
NCT01793168 -
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
|
||
Active, not recruiting |
NCT05826171 -
Priming Motor Learning Through Exercise in People With Spinocerebellar Ataxia
|
N/A | |
Recruiting |
NCT04399265 -
Efficacy Of Oral Trehalose In Spinocerebellar Ataxia 3
|
N/A | |
Completed |
NCT03885167 -
Identification of Biomarkers in Spinocerebellar Ataxia 3
|
||
Terminated |
NCT05160558 -
A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3
|
Phase 1 | |
Recruiting |
NCT05822908 -
A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD
|
Phase 1/Phase 2 | |
Not yet recruiting |
NCT03378414 -
Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar Ataxia
|
Phase 2 | |
Completed |
NCT05502432 -
Repetitive Transcranial Magnetic Stimulation in SCA3 Patients
|
N/A | |
Recruiting |
NCT04714307 -
Neuropsychiatry and Cognition in SCA3/MJD
|
||
Recruiting |
NCT05557786 -
Treatment of Transcranial Alternating Current Stimulation(tACS)on Cerebellar Ataxia
|
||
Recruiting |
NCT01060371 -
Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias
|