- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00803868
Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 3
Contacts and Locations
Study Locations
-
-
Florida
-
Tampa, Florida, United States, 33612
- University of South Florida
-
-
Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- The Children's Hospital of Philadelphia
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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).
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.
Exclusion Criteria:
- 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.
- 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).
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: 2
Placebo
|
placebo matching study drug up to 1mg po bid
|
Experimental: 1
Varenicline
|
up to 1mg po bid for 9 weeks
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Friedreich Ataxia Rating Scale (FARS)
Time Frame: 9 weeks
|
9 weeks
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Theresa Zesiewicz, M.D., University of South Florida
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neurologic Manifestations
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Dyskinesias
- Spinal Cord Diseases
- Heredodegenerative Disorders, Nervous System
- Mitochondrial Diseases
- Cerebellar Diseases
- Spinocerebellar Degenerations
- Ataxia
- Cerebellar Ataxia
- Friedreich Ataxia
- Physiological Effects of Drugs
- Neurotransmitter Agents
- Molecular Mechanisms of Pharmacological Action
- Cholinergic Agents
- Nicotinic Agonists
- Cholinergic Agonists
- Varenicline
Other Study ID Numbers
- 6
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Friedreich's Ataxia
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University of ChicagoPfizer; Biogen; APDM Wearable TechnologiesActive, not recruitingSpinocerebellar Ataxia Type 3 | Friedreich Ataxia | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6United States
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University of South FloridaCompletedFriedreich's Ataxia | Spinocerebellar Ataxia - All Sub-typesUnited States
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Santhera PharmaceuticalsCompletedFreidreich's AtaxiaGermany, Netherlands, France, Austria, Belgium
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Institut National de la Santé Et de la Recherche...Not yet recruiting
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PTC TherapeuticsEnrolling by invitationFriedreich AtaxiaUnited States, Australia, Brazil, Canada, France, Germany, Italy, New Zealand, Spain
-
PTC TherapeuticsActive, not recruitingFriedreich AtaxiaUnited States
-
PTC TherapeuticsCompletedFriedreich AtaxiaUnited States, Australia, Brazil, Canada, France, Germany, Italy, New Zealand, Spain
-
Children's Hospital of PhiladelphiaFriedreich's Ataxia Research Alliance; Stealth BioTherapeutics Inc.Active, not recruitingFriedreich AtaxiaUnited States
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Metro International Biotech, LLCChildren's Hospital of PhiladelphiaCompleted
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Santhera PharmaceuticalsCompletedFriedreich's AtaxiaUnited States
Clinical Trials on varenicline
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Arizona State UniversityNational Institute on Drug Abuse (NIDA); Pfizer; University of Nevada, Reno; Los...CompletedSmoking Cessation | Withdrawal Symptoms | Tobacco DependenceUnited States
-
PfizerWithdrawn
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University of California, Los AngelesNational Institute on Drug Abuse (NIDA)CompletedMethamphetamine Addiction | Crystal Meth Addiction | Amphetamine AddictionUnited States
-
Korea University Anam HospitalCompletedHealthyKorea, Republic of
-
Butler HospitalBrown UniversityCompletedDepressive Disorder | SmokingUnited States
-
Medical University of South CarolinaCompletedSmoking | Smoking Cessation | Smoking, Tobacco | Smoking, CigaretteUnited States
-
University of Wisconsin, MadisonNational Cancer Institute (NCI)Enrolling by invitation
-
PfizerCompletedSmoking CessationUnited States, Korea, Republic of, Taiwan, Canada, Georgia, Russian Federation
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Oyster Point Pharma, Inc.CompletedHealthy SubjectsUnited States