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Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia

Primary Purpose

Friedreich's Ataxia

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
varenicline
placebo
Sponsored by
University of South Florida
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Friedreich's Ataxia

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Outpatients with FA diagnosed by confirmed by genetic testing.
  2. Age 18 years to 80 years.
  3. 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.
  4. 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

  5. Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
  6. Patient permission (informed consent).
  7. 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:

  1. Any unstable illness that in the investigator's opinion preclude participation in this study.
  2. Use of another investigational product within the past 28 days.
  3. Patients with a history of substance abuse.
  4. Presence of preexisting psychiatric illness (specifically schizophrenia, bipolar disorder, or history of suicide attempt).
  5. Presence of diabetes (as determined by fasting blood glucose labs within the past 6 months).
  6. 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.
  7. 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.
  8. Concurrent treatment with any MAOIs, Wellbutrin, or nicotine patches.
  9. 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).
  10. Dementia or other psychiatric illness that prevents the patient from giving informed consent (MMSE less than 25).
  11. Legal incapacity or limited legal capacity.
  12. 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).
  13. Abnormal WBC hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).

Sites / Locations

  • University of South Florida
  • The Children's Hospital of Philadelphia

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

1

2

Arm Description

Varenicline

Placebo

Outcomes

Primary Outcome Measures

Friedreich Ataxia Rating Scale (FARS)

Secondary Outcome Measures

Full Information

First Posted
December 4, 2008
Last Updated
July 23, 2012
Sponsor
University of South Florida
Collaborators
Friedreich's Ataxia Research Alliance, Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT00803868
Brief Title
Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia
Official Title
Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia
Study Type
Interventional

2. Study Status

Record Verification Date
July 2012
Overall Recruitment Status
Terminated
Why Stopped
The DSMB recommended that the study be stopped as a result of concerns regarding safety and intolerability and insufficient evidence of efficacy.
Study Start Date
May 2009 (undefined)
Primary Completion Date
May 2010 (Actual)
Study Completion Date
May 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of South Florida
Collaborators
Friedreich's Ataxia Research Alliance, Pfizer

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to determine if varenicline is effective in treating symptoms of Friedreich's ataxia.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Friedreich's Ataxia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
28 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Experimental
Arm Description
Varenicline
Arm Title
2
Arm Type
Placebo Comparator
Arm Description
Placebo
Intervention Type
Drug
Intervention Name(s)
varenicline
Intervention Description
up to 1mg po bid for 9 weeks
Intervention Type
Drug
Intervention Name(s)
placebo
Intervention Description
placebo matching study drug up to 1mg po bid
Primary Outcome Measure Information:
Title
Friedreich Ataxia Rating Scale (FARS)
Time Frame
9 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Theresa Zesiewicz, M.D.
Organizational Affiliation
University of South Florida
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States

12. IPD Sharing Statement

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Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia

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