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Study to Identify Clinical, Imaging and Biologic Markers of Parkinson Disease Progression (PPMI)

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2013 by Michael J. Fox Foundation for Parkinson's Research
Sponsor:
Collaborator:
Institute for Neurodegenerative Disorders
Information provided by (Responsible Party):
Ken Marek, MD, Michael J. Fox Foundation for Parkinson's Research
ClinicalTrials.gov Identifier:
NCT01141023
First received: June 8, 2010
Last updated: October 23, 2013
Last verified: October 2013
  Purpose

This is a observational, multi-center study to assess progression of clinical features, imaging and biologic biomarkers in Parkinson disease (PD) patients compared to healthy controls (HC) and in PD patient subtypes.

The primary objective of this study is to identify clinical, imaging and biologic markers of PD progression for use in clinical trials of disease-modifying therapies.


Condition Intervention Phase
Parkinson Disease
Drug: Datscan and AV-133
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Diagnostic
Official Title: The Parkinson's Progression Markers Initiative (PPMI)

Resource links provided by NLM:


Further study details as provided by Michael J. Fox Foundation for Parkinson's Research:

Primary Outcome Measures:
  • The mean rates of change and the variability around the mean of clinical, imaging and biomic outcomes in early PD patients, and the comparison of these rates between PD patient subsets and between PD, SWEDD, Prodromal and healthy subjects. [ Time Frame: Study intervals from 3 months - 36 months ] [ Designated as safety issue: No ]
    Specific examples of outcomes include MDS-UPDRS, dopamine transporter striatal uptake, vesicular monoamine transporter type-2 uptake, and serum and CSF alpha-synuclein. PD patient subsets maybe defined by baseline assessments, progression milestones and/or rate of clinical,imaging, or biomic change.


Secondary Outcome Measures:
  • Comparison between the rates of change in the mean of clinical, imaging and biomic outcomes in early PD patients and between PD and healthy subjects [ Time Frame: Study Intervals from 3 months to 36 months ] [ Designated as safety issue: No ]
  • Prevalence of measures of clinical, imaging and biomic outcomes in early PD patients and healthy subjects. [ Time Frame: from baseline to 36 months. ] [ Designated as safety issue: No ]
  • To establish the predictive value of early clinical non-motor features, baseline imaging and biomic outcomes for future course of disease. [ Time Frame: No time frame needed. ] [ Designated as safety issue: No ]
  • Exploratory analysis of comparison between the rates of change in the mean of clinical, imaging and biomic outcomes in prodromal PD with baseline DaTSCAN binding showing minimal to moderate DAT deficit and early PD patients and healthy subjects. [ Time Frame: Study intervals from 3 months to 36 months ] [ Designated as safety issue: No ]
  • Exploratory analysis of prevalence of measures of clinical, imaging and biomic outcomes in prodromal PD compared to early PD patients and healthy subjects [ Time Frame: Study intervals from baseline to 36 months ] [ Designated as safety issue: No ]
  • To examine the proportion of Prodromal subjects with one or more risk characteristics. [ Time Frame: No Time Frame needed ] [ Designated as safety issue: No ]
    To examine the proportion of Prodromal subjects with one or more risk characteristics [hyposmia (<10th percentile by age and gender), RBD, or LRRK2 mutation, and baseline DaTSCAN binding showing minimal to moderate DAT deficit] who phenoconvert within two years. To correlate the baseline DaTSCAN binding with risk of phenoconversion.

  • To conduct exploratory analyses to examine whether the progression of clinical, imaging, and biospecimen biomarkers will predict those subjects likely to phenoconvert. [ Time Frame: No Time frame needed ] [ Designated as safety issue: No ]
    For example, dopamine transfer loss during the prodromal period either independently or in combination with other biomarkers may provide a quantitative outcome associated with prodromal disease progression to phenoconversion.

  • To examine the proportion of SWEDD subjects that have a change in their clinical management at 24 months (SWEDD Clinical Diagnosis and Management Questionnaire). [ Time Frame: No Time Frame Needed ] [ Designated as safety issue: No ]
  • To conduct exploratory analyses in SWEDD subjects to examine the prevalence of measures of clinical, imaging, and biomic outcomes from baseline to 24 months. [ Time Frame: Interval 3 months to 24 months ] [ Designated as safety issue: No ]
    Examine the mean rates of change and the variability among these outcomes from 3 months to 24 months, and to examine the correlations between the rates of change in these outcomes from 3 months to 24 months.

  • To conduct exploratory analyses to determine whether the measures and change over time in clinical, imaging, and biomic outcomes are similar among the SWEDD, Prodromal, and PD subjects. [ Time Frame: No Time Frame Needed ] [ Designated as safety issue: No ]

Estimated Enrollment: 680
Study Start Date: June 2010
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: September 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Datscan and AV-133 Drug: Datscan and AV-133

Detailed Description:

PPMI will be a five-year natural history study (a minimum of 3-year involvement for each subject) of de novo idiopathic PD patients and healthy controls. This study will also include a SWEDD (subjects without evidence of dopaminergic deficit)and Prodromal populations.

All subjects will be comprehensively assessed at baseline and every three to six months thereafter. Subjects will undergo clinical (motor, neuropsychiatric and cognitive) and imaging assessments and will donate blood, urine, and cerebral spinal fluid (CSF). A blood sample for DNA will be collected. Data will be collected by each site under uniformly established protocols and data will be analyzed and stored at designated core facilities.

  Eligibility

Ages Eligible for Study:   30 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

Parkinson Disease (PD) Subjects:

  • A diagnosis of Parkinson disease for 2 years or less at Screening.
  • Confirmation from imaging core that screening DAT scan is consistent with dopamine transporter deficit, or if applicable a VMAT-2 PET scan consistent with vesicular monoamine transporter deficit.
  • Not expected to require PD medication with at least 6 months from Baseline.
  • Male or female age 30 years or older at time of PD diagnosis.

Healthy Control (HC) Subjects:

• Male or female age 30 years or older at Screening.

Exclusion Criteria:

Parkinson Disease (PD) Subjects:

  • Currently taking levodopa, dopamine agonists, MAO-B inhibitors, amantadine or other PD medication.
  • Has taken levodopa, dopamine agonists, MAO-B inhibitors or amantadine within 60 days of Baseline.
  • Has taken levodopa or dopamine agonists prior to Baseline for more than a total of 60 days.
  • Received any of the following drugs that might interfere with DAT imaging: Neuroleptics, metoclopramide, alpha methyldopa, methylphenidate, reserpine, or amphetamine derivative, within 6 months of Screening.
  • Current treatment with anticoagulants (e.g., coumadin, heparin) that might preclude safe completion of the lumbar puncture.

If applicable, currently taking medications that are known to cause QT-prolongation, or are currently taking tetrabenazine (TBZ or amphetamine type medications.

  • Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia.
  • Use of investigational drugs within 60 days prior to Baseline (dietary supplements taken outside of a clinical trial are not exclusionary, e.g., coenzyme Q10).

Healthy Control (HC) Subjects:

  • Current or active neurological disorder.
  • First degree relative with idiopathic PD (parent, sibling, child).
  • MoCA score < 26.
  • Received any of the following drugs that might interfere with DAT imaging: Neuroleptics, metoclopramide, alpha methyldopa, methylphenidate, reserpine, or amphetamine derivative, within 6 months of Screening.

If applicable, currently taking medications that are known to cause QT-prolongation, or are currently taking tetrabenazine (TBZ) or amphetamine type medications.

  • Current treatment with anticoagulants (e.g., coumadin, heparin) that might preclude safe completion of the lumbar puncture.
  • Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia.
  • Use of other investigational drugs within 60 days prior to baseline (dietary supplements taken outside of a clinical trial are not exclusionary, e.g., coenzyme Q10).

SWEDD Subjects:

All PD criteria apply, as above, except a SWEDD subject must have confirmation from imaging core that screening dopamine transporter SPECT scan shows no evidence of dopamine transporter deficit or if applicable a VMAT-2 PET scan shows no evidence of vesicular monoamine transporter deficit.

Prodromal Subjects:

Inclusion Criteria (Prodromal Subjects) 4.2.7.1. Subjects must have at least one of the following characteristics:

Hyposmia:

  1. Male or female age 60 years or older
  2. Confirmation from olfactory core that olfaction as determined by UPSIT is at or below the 10th percentile by age and gender

REM Behavior Disorder (RBD):

  1. Male or female age 60 years or older
  2. Confirmation from sleep core that subject's Polysomnography (PSG) meets criteria for RBD

LRRK2:

  1. Male or female age 60 years or older
  2. Written confirmation or documentation from testing facility that the individual is LRRK2 mutation positive 4.2.7.2. Confirmation from imaging core that screening dopamine transporter SPECT scan is read as eligible (see below). About 80 subjects will have a range of DAT deficit similar to subjects with early PD (mild to moderate DAT deficit). About 20 subjects will be selected with no DAT deficit or minimal DAT deficit similar in age, gender, and risk profile to those with mild to moderate DAT deficit. 4.2.7.3. Ability to provide written informed consent in accordance with Good Clinical Practice (GCP), International Conference on Harmonization (ICH), and local regulations. 4.2.7.4. Willing and able to comply with scheduled visits, required study procedures and laboratory tests. 4.2.7.5. Women may not be pregnant, lactating or planning pregnancy during the course of the study. Includes a negative urine pregnancy test on day of screening scan prior to injection (DaTSCAN).

Exclusion Criteria (Prodromal Subjects)

  1. Current or active clinically significant neurological disorder or psychiatric disorder (in the opinion of the Investigator).
  2. GDS score greater than or equal to 10 (GDS score of 5 - 9 requires Investigator discretion to enter study).
  3. STAI Form Y-1 greater than or equal to 54 requires Investigator discretion to enter study.
  4. A clinical diagnosis of dementia63 as determined by the investigator (Appendix 1).
  5. A clinical diagnosis of Parkinson disease at the Screening visit as determined by the Investigator.
  6. Received any of the following drugs that might interfere with dopamine transporter SPECT imaging: Neuroleptics, metoclopramide, alpha methyldopa, methylphenidate, reserpine, or amphetamine derivative, within 6 months of Screening.
  7. Current treatment with anticoagulants (e.g., coumadin, heparin) that might preclude safe completion of the lumbar puncture.
  8. Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia.
  9. Any other medical or psychiatric condition or lab abnormality, which in the opinion of the investigator might preclude participation.
  10. Use of investigational drugs or devices within 60 days prior to Baseline (dietary supplements taken outside of a clinical trial are not exclusionary, e.g., coenzyme Q10).
  11. Previously obtained MRI scan with evidence of clinically significant neurological disorder (in the opinion of the Investigator).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01141023

  Hide Study Locations
Locations
United States, Alabama
University of Alabama at Birmingham Recruiting
Birmingham, Alabama, United States, 35294
Contact: Stephanie Guthrie    205-996-4033    slguth@uab.edu   
Principal Investigator: David G. Standaert, MD PHD         
Sub-Investigator: Victor Sung, MD, PhD         
United States, Arizona
Banner Research Institute Recruiting
Sun City, Arizona, United States, 85351
Contact: Lauren Arnieri    623-875-6521    lauren.arnieri@bannerhealth.com   
Contact: Sanja Obradov, BA    623-876-5468    sanja.obradov@sunhealth.org   
Principal Investigator: Holly Shill, MD         
Sub-Investigator: Charles Adler, MD PhD         
United States, California
University of California San Diego Recruiting
La Jolla, California, United States, 92093-0948
Contact: Deborah Fontaine, RNCS MS    858-622-5800    dfontaine@ucsd.edu   
Principal Investigator: Douglas Galasko, MD         
Sub-Investigator: Stephanie Lessig, MD         
Sub-Investigator: Irene Litvan, MD         
The Parkinson's Institute Recruiting
Sunnyvale, California, United States, 94085
Contact: Linda Rees, MPH    408-542-5664    lrees@thepi.org   
Contact: Sakunthala Sundarrajan, BSD    408-542-5668    ssundarrajan@thepi.org   
Principal Investigator: Caroline M. Tanner, MD, PhD         
Sub-Investigator: Melanie Brandabur, MD, MDS         
Sub-Investigator: Grace Liang, MD         
Sub-Investigator: James Tetrud, MD         
United States, Connecticut
Institute For Neurodegenerative Disorders Recruiting
New Haven, Connecticut, United States, 06510
Contact: Laura Leary, BS    203-401-4332    lleary@indd.org   
Contact: Pam Becker, RN    203-401-4344    pbecker@indd.org   
Principal Investigator: Danna L Jennings, MD         
Sub-Investigator: David Russell, MD         
United States, Florida
Parkinson's Disease& Movement Disorder Center of Boca Raton Recruiting
Boca Raton, Florida, United States, 33486
Contact: Jennifer Bar-nur, BS    561-392-1818 ext 6    jbar-nur@parkinsonscenter.org   
Contact: Angela James    561-392-1818 ext 6    ajames@parkinsonscenter.org   
Principal Investigator: Stuart Isaacson, MD         
University of South Florida Recruiting
Tampa, Florida, United States, 33606
Contact: Laura Murray, LPN    813-369-0764    lmurray1@health.usf.edu   
Contact: Patti Lowe, LPN    813-844-4455    plowe@health.usf.edu   
Principal Investigator: Robert Hauser, MD         
Sub-Investigator: Deborah Burke, MD         
United States, Georgia
Emory University School of Medicine Recruiting
Atlanta, Georgia, United States, 30329
Contact: Rebecca McMurray, RN, BS    404-728-6427    rmcmurra@emory.edu   
Principal Investigator: Stewart A Factor, DO         
United States, Illinois
Northwestern University Recruiting
Chicago, Illinois, United States, 60611
Contact: Karen Williams    312-503-5645    k-williams8@northwestern.edu   
Contact: Christina Warner, BA    312-503-1519    christina.warner@northwestern.edu   
Principal Investigator: Tanya Simuni, MD         
Sub-Investigator: Aleksandar Videnovic, MD         
Sub-Investigator: Cindy Zadikoff, MD FRCPC         
United States, Maryland
John Hopkins University Recruiting
Baltimore, Maryland, United States, 21287
Contact: Arita McCoy, RN    410-955-8795    amccoy6@jhmi.edu   
Sub-Investigator: Ted Dawson, MD PhD         
Principal Investigator: Zoltan Mari, MD         
United States, Massachusetts
Boston University Recruiting
Boston, Massachusetts, United States, 02118
Contact: Cathi Thomas, RN MS    617-638-7737    neurocat@bu.edu   
Contact: Ray C. James    617 638-7745    rcjames@bu.edu   
Principal Investigator: Samual Frank, MD         
Sub-Investigator: Marie H. Saint-Hilaire, MD         
United States, New York
University of Rochester Recruiting
Rochester, New York, United States, 14620
Contact: Cheryl Deeley, MS RNC    585-341-7515    ppmi@urmc.rochester.edu   
Contact: Courtney Bishop, BS, MPH    585-341-7500    ppmi@urmc.rochester.edu   
Principal Investigator: Irene Richards, MD         
United States, Ohio
University of Cincinnati/Cincinnati Children's Hospital Recruiting
Cincinnati, Ohio, United States, 45219
Contact: Kristy Sullivan    513-558-6517    kristy.sullivan@uc.edu   
Contact: Angela Molloy, RN    513-558-7118    englanar@ucmail.uc.edu   
Principal Investigator: Alberto Espay, MD, MSC         
Sub-Investigator: Andrew Duker, MD         
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
Contact: Jennifer Mule, BS    216-444-1134    mulej@ccf.org   
Contact: Nancy Monahan    216-445-5637    monahan@ccf.org   
Principal Investigator: Hubert H. Fernandez, MD         
Sub-Investigator: Anwar Ahmed, MD         
Sub-Investigator: Ilia Itin, MD         
Sub-Investigator: Michael Gostkowski, DO         
United States, Oregon
Oregon Health &Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Emily Pighetti, MA    503-494-1382    pighetti@ohsu.edu   
Contact: Anna Lovelace, BS, BA    503-494-9531    lovelaca@ohsu.edu   
Principal Investigator: Penelope Hogarth, MD         
United States, Pennsylvania
University of Pennsylvania Recruiting
Pennsylvania, Pennsylvania, United States, 19107
Contact: Abigail Darin    215-829-7374    abigail.darin@uphs.upenn.edu   
Contact: Baochan Tran    215 829 7104    boachant@uphs.upenn.edu   
Principal Investigator: Matthew Stern, MD         
Sub-Investigator: Lama Chahine, MD         
Sub-Investigator: Rizwan Akhtar, MD,PhD         
United States, Texas
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: Christine Hunter, RN    713-798-3951    chunter@bcm.edu   
Principal Investigator: Joseph Jankovic, MD         
Sub-Investigator: Joohi Shahed, MD         
United States, Washington
Univ of Washington and VA Puget Sound Health Care System Recruiting
Seattle, Washington, United States, 98104
Contact: Marne Baca    206-277-6977    marneb@u.washington.edu   
Contact: Heli Venkov, BS    206-277-5087    heli.venkov@staff.siber.org   
Principal Investigator: Jim Leverenz         
Sub-Investigator: Veronica Hicks         
Sub-Investigator: Gretchen Todd         
Australia
Macquarie University Recruiting
Sydney, Australia, NSW2109
Contact: Madelaine Ranola    +61-298-123-720    madelaine.ranola@me.com   
Principal Investigator: Dominic Rowe         
Sub-Investigator: Carolyn Orr, MD         
Austria
Innsbruck Medical University Recruiting
Innsbruck, Austria, 6020
Contact: Fabienne Sprenger    43 512 504 81553    fabienne.sprenger@i-med.ac.at   
Sub-Investigator: Philipp Mahlknecht, MD         
Principal Investigator: Werner Poewe, MD         
Sub-Investigator: Klaus Seppi, MD         
Sub-Investigator: Sabine Spielberger         
Sub-Investigator: Eva Reiter, MD         
Germany
Paracelsus-Elena Klinik Recruiting
Kassel, Germany, 34128
Contact: Diana Willeke    49 561 6009 272    diana.willeke@pk-mx.de   
Contact: Manuela Wilfing    49 561 6009-272    manuela.wilfling@pk-mx.de   
Sub-Investigator: Jens Ebentheuer         
Principal Investigator: Brit Mollenhauer, MD         
University of Tuebingen Recruiting
Tuebingen, Germany, 72076
Contact: Katharina Gauss    +49 7071 2983272    katharina.gauss@med.uni-tuebingen.de   
Contact: Alexandra Gaenslen    +49 7072 2980171    alexandra.gaenslen@klinikum.uni-tuebingen.de   
Principal Investigator: Daniela Berg, MD         
Sub-Investigator: Walter Maetzler         
Sub-Investigator: Isabel Wurster         
Sub-Investigator: Kathrin Brockmann, MD         
Sub-Investigator: Karin Srulijes         
Italy
Universita Federico II Recruiting
Napoli, Italy, 80131
Contact: Susan Ainscough, BA    39 340 519 2659    ricercaparkinson@gmail.com   
Principal Investigator: Paolo Barone, MD, PhD         
Sub-Investigator: Autilia Cozzolino, MD         
Sub-Investigator: Alfonso Mauro         
Sub-Investigator: Giampiero Volpe, MD         
United Kingdom
Imperial College London Recruiting
London, United Kingdom, W12 0NN
Contact: Bina Shah, BSC    44 203-313 0622    b.shah@imperial.ac.uk   
Principal Investigator: David Brooks, MD         
Sub-Investigator: Sophie Molloy, MD         
Sub-Investigator: Nicola Pavese, MD         
Sponsors and Collaborators
Ken Marek, MD
Institute for Neurodegenerative Disorders
Investigators
Study Chair: Kenneth L Marek, MD Institute for Neurodegenerative Disorders
Principal Investigator: John Q. Trojanowski, MD, PhD University of Pennsylvania
Principal Investigator: Arthur W. Toga, PhD University of California, Los Angeles
Principal Investigator: Alison Ansbach, MS Coriell Institute for Medical Research
Principal Investigator: Karl Kieburtz, MD Clinical Trials Coordination Center
Principal Investigator: Andrew Singleton, PhD Laboratory of Neurogenetics; National Institute on Aging NIH
Principal Investigator: John P Seibyl, MD Institute for Neurodegenerative Disorders
Principal Investigator: Christopher Coffey, PhD Clinical Trials Statistical and Data Management Center, University of Iowa
  More Information

No publications provided

Responsible Party: Ken Marek, MD, Study Chair, Michael J. Fox Foundation for Parkinson's Research
ClinicalTrials.gov Identifier: NCT01141023     History of Changes
Other Study ID Numbers: PPMI-001
Study First Received: June 8, 2010
Last Updated: October 23, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Michael J. Fox Foundation for Parkinson's Research:
Parkinson
Bio-markers
Neurodegenerative disorder
Imaging
Prodromal

Additional relevant MeSH terms:
Parkinson Disease
Disease Progression
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases
Disease Attributes
Pathologic Processes

ClinicalTrials.gov processed this record on August 20, 2014