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Efficacy, Safety and Tolerability of PF-06649751 in Parkinson's Disease Patients at Early Stage of the Disease

Phase 2
Terminated
Conditions
Parkinson Disease
Interventions
Drug: Placebo
Drug: PF-06649751
Registration Number
NCT02847650
Lead Sponsor
Pfizer
Brief Summary

The purpose of this study is to evaluate the efficacy, safety and tolerability of PF-06649751 in Parkinson's disease patients at early stage of the disease.

Detailed Description

The B7601011 study has a randomized, double-blind, placebo-controlled parallel group design. Approximately 88 subjects will be randomized to 2 treatment groups. Each subject will participate in the study for approximately 23 weeks including a 30 day screening period, 15 week double blind treatment period, and an approximately 28 day follow-up period.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
57
Inclusion Criteria
  • Females of non-childbearing potential and/or male subjects
  • Clinical diagnosis of Parkinson's disease.
  • Parkinson's Disease Hoehn & Yahr Stage I-III inclusive
  • Treatment naïve or history of prior incidental treatment with dopaminergic agents for no more than 28 days
  • Able to refrain from any Parkinson's disease medication not permitted by the protocol.
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Exclusion Criteria
  • History or presence of atypical Parkinsonian syndrome.
  • Severe acute or chronic medical or psychiatric condition or cognitive impairment or laboratory abnormality.
  • Any condition possibly affecting drug absorption.
  • Participation in other studies involving investigational drug(s), or treatment with any investigational drug within 30 days.
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Study & Design

Study Type
INTERVENTIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
PlaceboPlacebo-
PF-06649751PF-06649751-
Primary Outcome Measures
NameTimeMethod
Change From Baseline in the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III Total Score at Week 15Baseline (Day -1/randomization), Week 15

MDS-UPDRS Part III was used to assess the motor signs of Parkinson's disease. It was comprised of 33 sub-scores based on 18 items, several with right, left or other body distribution scores. Each question was anchored with 5 responses that were linked to commonly accepted clinical terms: 0=normal, 1=slight, 2=mild, 3=moderate, and 4=severe. The MDS-UPDRS Part III total score range is 0-132. Higher score indicates more severe motor signs of Parkinson's disease. A negative change from baseline represents an improvement in motor function.

Secondary Outcome Measures
NameTimeMethod
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)From first dose of study treatment up to 28 days after last dose (up to Day 133)

An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; life-threatening (immediate risk of death); initial or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect. Treatment-emergent AEs were those with initial onset or increasing in severity after the first dose of study treatment.

Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality)Baseline (Day -1/randomization) up to Day 119 follow-up visit

Following safety laboratory parameters were assessed against pre-defined abnormality criteria: hematology (hemoglobin, hematocrit, red blood cell count, mean corpuscular volume, mean corpuscular hemoglobin, mean corpuscular hemoglobin concentration, platelet count, white blood cell count, absolute total neutrophils, absolute eosinophils, absolute basophils, absolute monocytes, and absolute lymphocytes); chemistry (blood urea nitrogen/urea and creatinine, glucose , calcium, sodium, potassium, chloride, total bicarbonate, aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\], total bilirubin, alkaline phosphatase, uric acid, albumin, total protein); urinalysis (pH, qualitative glucose, qualitative protein, qualitative blood, ketones, nitrites, leukocyte esterase, urine bilirubin, urobilinogen, urine creatinine, microscopy, and specific gravity).

Number of Participants With Worsening and New Onset Suicidality as Assessed by Columbia Suicide Severity Rating Scale (C-SSRS)Baseline (Day -1/randomization) up to Day 119 follow-up visit

The C-SSRS is an interview based rating scale to systematically assess suicidal ideation and suicidal behavior. C-SSRS responses were mapped to the Columbia Classification Algorithm of Suicide Assessment (C-CASA). Participants with new onset suicidality were those without suicidal ideation and behavior at baseline and reported any suicidal behavior or ideation post-baseline as assessed by C-CASA code mapped from C-SSRS data. Participants with worsening suicidality were those who moved to a lower numbered C-CASA category than was reported at baseline.

Change From Baseline in Questionnaire for Impulsive-Compulsive Disorders in Parkinson's Disease - Rating Scale (QUIP-RS) Total Score at Days 35, 63, and 105Baseline (Day -1 or randomization); Days 35, 63, 105

The QUIP-RS has 4 primary questions pertaining to commonly reported thoughts, urges/desires, and behaviors associated with impulsive-compulsive disorder , each applied to the 4 impulsive-compulsive disorders (compulsive gambling, buying, eating, and sexual behavior) and 3 related disorders (medication use, punding, and hobbyism). Each question is anchored with the following 5 responses: Never (0), Rarely (1), Sometimes (2), Often (3), and Very Often (4). The scoring range for each item (ie, disorder) is 0-16. The QUIP-RS total score range is 0-64. Higher score indicates a greater level of the impulsive compulsive disorder.

Total Physician Withdrawal Checklist (PWC-20) ScoreDay 119

The PWC-20 is a 20-item reliable and sensitive instrument for the assessment of benzodiazepine-like discontinuation symptoms. The total PWC-20 score is the sum of 20 item scores and ranges between 0 and 60. The higher score indicates more frequent/severe symptoms.

Number of Participants With Vital Signs Data Meeting Categorical Summarization and Orthostatic Hypotension CriteriaBaseline (Day -1/randomization) up to Day 119 follow-up visit

Vital signs categorical summarization criteria: 1) supine and standing systolic blood pressure (SBP) \<90 millimeters of mercury (mmHg); 2) supine and standing diastolic blood pressure (DBP) \<50 mmHg; 3) supine pulse rate \<40 or \>120 beats per minute (bpm); 4) standing pulse rate \<40 or \>140 bpm; 5) maximum change from baseline (increase or decrease) in supine and standing DBP greater than or equal to (\>=) 20 mmHg; 6) maximum change from baseline (increase or decrease) in supine and standing SBP \>=30 mmHg. Orthostatic hypotension criterion was defined as a decrease of \>=20 mmHg for SBP or \>=10 mmHg for DBP 2 minutes after standing from a supine position.

Number of Participants Meeting the Categorical Summarization Criteria for Electrocardiogram (ECG) ParametersBaseline (Day -1/randomization) up to Day 119 follow-up visit

ECG categorical summarization criteria: 1) QRS duration (time from ECG Q wave to the end of the S wave corresponding to ventricle depolarization): \>=140 milliseconds (msec), \>=50% increase from baseline; 2) PR interval (the interval between the start of the P wave and the start of the QRS complex, corresponding to the time between the onset of the atrial depolarization and onset of ventricular depolarization): \>=300 msec, \>=25% increase when baseline is \> 200 msec or \>=50% increase when baseline is less than or equal to (\<=) 200 msec; 3) QT interval (time from ECG Q wave to the end of the T wave corresponding to electrical systole): absolute value of \>=500 msec; 4) QTcF interval (QT corrected for heart rate using Fridericia's formula): absolute value of 450 to \<480 msec, 480 to \<500 msec, \>=500 msec; an increase from baseline of 30 to \<60 msec or \>=60 msec.

Trial Locations

Locations (37)

Parkinson's Disease and Movement Disorders Center of Boca Raton

🇺🇸

Boca Raton, Florida, United States

Asheville Neurology Specialists PA

🇺🇸

Asheville, North Carolina, United States

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Baylor College of Medicine

🇺🇸

Houston, Texas, United States

Brigham and Women's Hospital

🇺🇸

Boston, Massachusetts, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

St Joseph's Hospital and Medical Center, Barrow Neurology Clinics

🇺🇸

Phoenix, Arizona, United States

St. Joseph's Hospital and Medical Center

🇺🇸

Phoenix, Arizona, United States

Atlanta Center for Medical Research

🇺🇸

Atlanta, Georgia, United States

Rush University Medical Center

🇺🇸

Chicago, Illinois, United States

University of Toledo

🇺🇸

Toledo, Ohio, United States

University of Toledo, Gardner-McMaster Parkinson Center

🇺🇸

Toledo, Ohio, United States

AS Clinical Research Consultants of North Texas, PLLC

🇺🇸

Greenville, Texas, United States

Sentara Neurology Specialists

🇺🇸

Virginia Beach, Virginia, United States

Hopital Henri Mondor

🇫🇷

Créteil, France

Hôpital Henri Mondor

🇫🇷

CRÉTEIL Cedex, France

CHU de Grenoble Alpes

🇫🇷

Grenoble, France

CHU Grenoble Alpes

🇫🇷

La Tronche, France

Hospital de La Timone

🇫🇷

Marseille, France

Hopital Pitie Salpetriere

🇫🇷

Paris, France

Hospital La Timone

🇫🇷

Marseille, France

Hopital Pitie-Salpetriere

🇫🇷

Paris, France

St. Josef Hospital GmbH

🇩🇪

Bochum, Nordrhein-westfalen, Germany

Praxis Oehlwein Outpatient clinic for PD, DBS, Movement Disorders

🇩🇪

Gera, Germany

Klinik Haag i. OB

🇩🇪

Haag I. OB, Germany

Universitätsklinikum Gießen und Marburg GmbH

🇩🇪

Marburg, Germany

Paracelsus-Elena-Klinik Kassel

🇩🇪

Kassel, Germany

University hospital Tuebingen

🇩🇪

Tuebingen, Germany

Universitaetsklinik Ulm

🇩🇪

Ulm, Germany

University of Miami

🇺🇸

Miami, Florida, United States

Pharmacy, Edith Wolfson Medical Center

🇮🇱

Holon, Israel

Edith Wolfson Medical Center

🇮🇱

Holon, Israel

University of South Florida Parkinson's Disease and Movement Disorders Center

🇺🇸

Tampa, Florida, United States

University of South Florida Carol and Frank Morsani Center for Advanced Health Care

🇺🇸

Tampa, Florida, United States

University of South Florida Faculty Office Building

🇺🇸

Tampa, Florida, United States

University of South Florida

🇺🇸

Tampa, Florida, United States

University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

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