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Pioglitazone in Early Parkinson's Disease

Phase 2
Completed
Conditions
Parkinson's Disease
Interventions
Drug: placebo
Registration Number
NCT01280123
Lead Sponsor
University of Rochester
Brief Summary

This is a multi-center, double-blind, placebo controlled clinical trial of two dosages of oral pioglitazone (15 milligram(mg) and 45 milligram (mg)) for safety, tolerability, and futility.

Subjects who are on stable dose of rasagiline 1 mg/day or selegiline 10 mg/day for at least 8 weeks but no more than 8 months, will be randomized to one of two dosages of oral pioglitazone (15 mg and 45 mg) or matching placebo.

The study will measure disease progression by the change in total Unified Parkinson's Disease Rating Scale (UPDRS) score between the baseline visit and 44 weeks.

Detailed Description

A Multi-Center, Double-Blind, Placebo-Controlled Phase II Study of Pioglitazone in Early Parkinson's Disease (PD). The patient population has early stage PD (\< 5 years from diagnosis), must be treated with 1 mg/day of rasagiline or 10 mg/day of selegiline for at least 8 weeks but not more than 8 months prior to enrollment.

The primary objective of this clinical trial is to assess the futility of pioglitazone on PD disease progression as measured by the change in total UPDRS score between the baseline visit and 44 weeks. The secondary objectives of the study are to collect additional efficacy and safety/tolerability data to be used in planning a subsequent Phase III trial of pioglitazone in early, treated PD. Measures of cognition, mood and blood- and urine-based biomarkers will also be explored. Subjects in this trial are randomly assigned in a 1:1:1 ratio to one of three study arms: 15 mg, 45 mg or placebo.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
210
Inclusion Criteria
  1. Willing and able to give informed consent.
  2. Men and women with idiopathic PD of less than 5 years duration from diagnosis with a Hoehn and Yahr Stage < 2.
  3. On stable dosage of rasagiline 1 mg/day or selegiline 10 mg/day for at least 8 weeks but not more than 8 months prior to baseline. Expected to remain on stable dose of rasagiline or selegiline as the only treatment for their PD for the duration of the study (44 weeks).
  4. Diagnosis must be confirmed by bradykinesia plus one of the other cardinal signs (resting tremor, rigidity) being present, without any other known or suspected cause of parkinsonism. The clinical signs must be asymmetric.
  5. Subjects may be taking stable doses (30 days) of anticholinergics or creatine (< 5gm/day) but must be expected to remain on the same dose.
  6. Age > 30 years.
  7. Women who are not postmenopausal or surgically sterile must use a medically accepted contraceptive regimen for at least 60 days before the baseline visit, and agree to continue such use throughout the duration of the study and for 30 days after the final dose of study drug.
Exclusion Criteria
  1. Exposure to dopaminergic PD therapy or amantadine within 60 days prior to baseline visit or for 90 days or more at any point in the past
  2. Use of any of the following drugs within 180 days prior to baseline: neuroleptics, metoclopramide, alpha-methyldopa, clozapine, olanzapine and flunarizine.
  3. Use of any of the following drugs within 90 days prior to baseline: methylphenidate, cinnarizine, reserpine, tetrabenazine, amphetamine or monoamine oxidase (MAO)-A inhibitors (pargyline, phenelzine, and tranylcypromine).
  4. Presence of drug-induced parkinsonism (e.g., metoclopramide, flunarizine), metabolic identified neurogenetic disorders (e.g., Wilson's disease), encephalitis, or other atypical Parkinsonian syndromes (e.g., progressive supranuclear palsy, multiple system atrophy).
  5. Participation in other drug studies or receipt of other investigational drugs within 30 days prior to baseline or during the study.
  6. Presence of freezing.
  7. Any clinically significant psychiatric or medical condition or laboratory abnormality, which would in the judgment of the Investigator interfere with the subject's ability to participate.
  8. History of stereotaxic brain surgery for PD
  9. Clinically significant structural brain disease that the investigator believes would interfere with study evaluations.
  10. History of congestive heart failure.
  11. Use of pioglitazone or rosiglitazone within 90 days before randomization.
  12. Known intolerance to pioglitazone or rosiglitazone.
  13. Allergy to rasagiline or selegiline, or contraindication to rasagiline or selegiline use.
  14. Type I or Type II diabetes mellitus.
  15. HgbA1C greater than or equal to 6% at Screening.
  16. Known liver disease or elevation of AST or ALT greater than 2.5 times the upper limit of normal.
  17. Known history of osteoporosis. All women ≥ 65 years of age or men and woman at high risk of osteoporosis should have documented evidence of screening for osteoporosis. Factors associated with high risk of osteoporosis include: previous non traumatic fracture, chronic glucocorticoid use, body weight under 58 kg, family history of hip fracture, current cigarette smoking, and excessive alcohol intake.
  18. Drug or alcohol use or dependence that, in the opinion of the Investigator, would interfere with the safe conduct of the study.
  19. Significant peripheral edema (2+ or more) of the extremities of any etiology.
  20. Current or planned use of gemfibrozil or rifampin during the trial.
  21. History of bladder cancer.
  22. Evidence of hematuria which has not been evaluated for evidence of bladder cancer. (Documentation of work up or a repeat urine test that was negative for hematuria and the primary care physician or urologist does not feel that further work up is required.)
  23. History of macular edema.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
15 mg pioglitazonePioglitazone15 mg pioglitazone
45 mg pioglitazonePioglitazone45 mg pioglitazone
Matching PlaceboplaceboPlacebo
Primary Outcome Measures
NameTimeMethod
Change in Total Unified Parkinson's Disease Rating Scale (UPDRS) Score From Baseline to 44 Weeks44 weeks

Change in total UPDRS score from baseline to 44 weeks (in subjects treated with rasagiline 1 mg/day or selegiline 10 mg/day).

The Total UPDRS is the sum of parts I, II, and III. The possible range of the total UPDRS is from 0-176. Higher values indicate worse outcomes.

The change is 44 weeks - baseline.

Secondary Outcome Measures
NameTimeMethod
Change in Parkinson's Disease Questionnaire (PDQ-39) From Baseline to 44 Weeks44 weeks

The Parkinson's Disease Questionnaire (PDQ-39) is a short, 39 item measure of quality of life in subjects with Parkinson's disease. The questionnaire covers 8 aspects of quality of life: mobility, activities of daily living, emotional well-being, stigma, social support, cognitions, communication and bodily discomfort.

The total score ranges from 0 (never have difficulty) to 100 (always have difficulty). Lower scores reflect better quality of life.

Change in the 15-item Geriatric Depression Scale (GDS-15)From Baseline to 44 Weeks44 weeks

The Geriatric Depression Scale - 15 is a short 15 yes or no question instrument for assessing depression in the elderly. It has been found to be particularly useful in assessing depression in Parkinson's Disease. A score of 0 to 5 is normal. A score greater than 5 suggests depression.

Change in Ambulatory Capacity From Baseline to 44 Weeks44 weeks

This is the sum of the 5 UPDRS questions regarding ambulatory capacity: falling, freezing, walking, gait, postural stability.

Ambulatory Capacity is calculated as the sum of items 13-15, 29, 30 of the Unified Parkinson's Disease Rating Scale (UPDRS). It ranges from 0-20. Higher scores are worse. Change is 44 weeks - baseline.

Change in Schwab and England Scale From Baseline to 44 Weeks44 weeks

The modified Schwab and England Activities of Daily Living is a single question ranging from 0-100% with anchors for each 10% interval. Higher scores are better (100% completely independent- 0% vegetative).

Change in the Mattis Dementia Rating Scale (DRS-2)From Baseline to 44 Weeks44 weeks

The Mattis dementia rating scale is a psychometric instrument designed to assess the extent and nature of dementia. Mattis Dementia Rating scale (DRS-2) raw score is the sum of 5 raw sub-scores (attention has possible 37 points, initiation/perseveration has possible 37 points, construction has possible 6 points, conceptualization has possible 39 points, memory has possible 25 points). Total range is 0-144. Higher scores are better.

Trial Locations

Locations (38)

University of Southern California

🇺🇸

Los Angeles, California, United States

Johns Hopkins University

🇺🇸

Baltimore, Maryland, United States

The Parkinson's & Movement Disorder Institute

🇺🇸

Fountain Valley, California, United States

University of Texas Southwestern Medical Center

🇺🇸

Dallas, Texas, United States

Brigham & Women's Hospital

🇺🇸

Boston, Massachusetts, United States

Beth Israel Deaconess Medical Center

🇺🇸

Boston, Massachusetts, United States

University of Miami

🇺🇸

Miami, Florida, United States

University of California San Fransisco

🇺🇸

San Fransisco, California, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

Washington University

🇺🇸

St Louis, Missouri, United States

University of Vermont

🇺🇸

Burlington, Vermont, United States

Univeristy of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Barrow Neurological Institute

🇺🇸

Phoenix, Arizona, United States

University of Florida

🇺🇸

Gainsville, Florida, United States

Duke University

🇺🇸

Durham, North Carolina, United States

Oregon Health & Science University

🇺🇸

Portland, Oregon, United States

Vanderbilt University

🇺🇸

Nashville, Tennessee, United States

Struthers Parkinson's Center

🇺🇸

Golden Valley, Minnesota, United States

Univeristy of Colorado Denver

🇺🇸

Aurora, Colorado, United States

University of South Florida

🇺🇸

Tampa, Florida, United States

University of Michigan

🇺🇸

Ann Arbor, Michigan, United States

University of Kentucky

🇺🇸

Lexington, Kentucky, United States

SUNY Downstate Medical Center

🇺🇸

Brooklyn, New York, United States

University of Florida, Jacksonville

🇺🇸

Jacksonville, Florida, United States

Emory University School of Medicine

🇺🇸

Atlanta, Georgia, United States

Pacific Health Research & Education Institute

🇺🇸

Honolulu, Hawaii, United States

Medical College of Georgia

🇺🇸

Augusta, Georgia, United States

Northwestern University

🇺🇸

Chicago, Illinois, United States

Rush University Medical Center

🇺🇸

Chicago, Illinois, United States

LSU Health Science Center Shreveport

🇺🇸

Shreveport, Louisiana, United States

Dartmouth Hitchcock Medical Center

🇺🇸

Lebanon, New Hampshire, United States

Michigan State University

🇺🇸

East Lansing, Michigan, United States

North Shore - LIJ Health System

🇺🇸

Manhasset, New York, United States

Medical University of South Carolina

🇺🇸

Charleston, South Carolina, United States

University of Virginia

🇺🇸

Charlottesville, Virginia, United States

Ochsner Clinic Foundation

🇺🇸

New Orleans, Louisiana, United States

Thomas Jefferson University

🇺🇸

Philadelphia, Pennsylvania, United States

University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

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