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

NCT ID: NCT00803868

Last Updated: 2012-07-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

28 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-05-31

Study Completion Date

2010-05-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Friedreich's Ataxia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

1

Varenicline

Group Type EXPERIMENTAL

varenicline

Intervention Type DRUG

up to 1mg po bid for 9 weeks

2

Placebo

Group Type PLACEBO_COMPARATOR

placebo

Intervention Type DRUG

placebo matching study drug up to 1mg po bid

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

varenicline

up to 1mg po bid for 9 weeks

Intervention Type DRUG

placebo

placebo matching study drug up to 1mg po bid

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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).
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Friedreich's Ataxia Research Alliance

OTHER

Sponsor Role collaborator

Pfizer

INDUSTRY

Sponsor Role collaborator

University of South Florida

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Theresa Zesiewicz, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of South Florida

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

University of South Florida

Tampa, Florida, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

6

Identifier Type: -

Identifier Source: org_study_id