Study to Identify Clinical, Imaging and Biologic Markers of Parkinson Disease Progression (PPMI)
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|ClinicalTrials.gov Identifier: NCT01141023|
Recruitment Status : Recruiting
First Posted : June 10, 2010
Last Update Posted : August 15, 2019
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 or disease||Intervention/treatment||Phase|
|Parkinson Disease||Drug: DaTscan||Phase 2|
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Amendment 14 - 26-Mar-2018 Revised to reflect the implementation of PPMI Wearables and Sensor Study Companion Protocol
Amendment 13 - 20-Nov-2017 Extension of study duration until 2023 for all cohorts (except Genetic Registry).
Amendment 12 - 01-Jun-2017 Planned date of trial extended to September 30, 2020 Ceasing new enrollments into the Genetic Registry for LRRK2 and GBA subjects Added Sensor - PPMI Companion Study Allow for consent to share contact information with FOUND team at any visit. Incorporation of Parkinson's Disease Risk Factor Questionnaire (PD-RFQ) into FOUND.
Amendment 11 - 01-Apr-2016 Added the collection of peripheral blood mononuclear cells (PBMC) from blood samples at interim visits.
Addition of PPMI Pathology Core/PPMI Brain and Tissue Bank Addition of Gait Assessment Companion Study (Select PPMI sites - Genetic Cohort only) Testing for additional GBA mutations - previously testing involved testing only for the GBA N370S mutation; however, going forward, testing will include tests for additional GBA mutations that are identified as being associated with certain ancestry.
Amendment 10 - 05-Oct-2015 Extended study period for PD and Healthy Control subjects through 8 years. For Prodromal subjects (RBD and Hyposmic) added iPSC companion study.
Amendment 9 - 01-Nov-2014 Addition of GBA throughout to document additional testing for GBA mutation. Allocation of subjects in Genetic Cohort and Genetic Registry revised to account for inclusion of GBA subjects.
Amendment 8 - 12-May-2014 Companion Protocols: skin biopsy/stem cell; Amyloid Imaging 18F Florbetaben; Family History Sub-Study; FOUND in PPMI registry. More detailed description of new procedures including: Advance directive for clinical research participation; Extension of study period (for SWEDDs with positive scans at Yr. 2) by continuing or re-inviting to study and followed (as per PD subjects through Month 60); Objective Parkinson Disease Measurement (OPDM) finger tapping measurement.
Amendment 7 - 14-Oct-2013 Pre-Screening Prodromal - RBD clarifications Pre-Screening Genetic Cohort - clarified Assessments/tests clarified, including addition of consent (or withdrawal of consent, if applicable) for future contact about future studies and PD family history data collection for Genetic cohort subjects as selected visits.
Amendment 6 - 29-May-2013 Genetics Coordination Core is added to study. The GCC included Genetic Cohort PD Subjects, Genetic Cohort Unaffected Subjects and Genetic Registry Subjects.
Amendment 5 - 27-Nov-2012 Olfactory and RBD subjects added as the Prodromal cohort.
Amendment 4 - 30-Mar-2012 Addition of 18F-AV-133 VMAT-2 PET Imaging for participating U.S. sites and Australia. (Refer to 18F-AV-13-PPMI companion protocol).
Number of sites increased from 21 to 24. Addition of cognitive categorization and diagnostic features assessments.
Amendment 3 - 15-Jul-2011 Addition of SWEDD subjects to study design. Blood Sampling Advisement to collect research samples in a fasted state.
Amendment 2 - 19-May-2011 Changed visit window from 30 days to 45 days for Baseline visit to be completed.
Section on DAT and SPECT Imaging - section changed to account for subject travel to the Institute for Neurodegenerative Disorders to conduct SPECT scanning and injection of either DaTSCAN or BCIT.
Main Protocol - 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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||680 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||The Parkinson's Progression Markers Initiative (PPMI)|
|Study Start Date :||June 2010|
|Estimated Primary Completion Date :||December 2023|
|Estimated Study Completion Date :||December 2023|
Experimental: Datscan SPECT Imaging
Subjects will b injected with 3-5 mCi of dopamine transporter. Within a 4 hour (+/- 30 minutes) window following the injection, subjects will undergo SPECT imaging on the camera.
Other Name: Ioflupane
- Mean Rates of Change [ Time Frame: Baseline to 156 months ]The mean rates of change and the variability around the mean of clinical, imaging and biomic outcomes in early PD patients, and where appropriate the comparison of these rates between PD patient subsets and between various subsets (including, but not limited to: PD vs. healthy subjects, PD vs. SWEDD, PD vs. prodromal, PD with and without LRRK2, GBA or SNCA mutation, unaffected LRRK2, GBA or SNCA mutation carriers vs. healthy controls) at study intervals ranging from 3 months to 36 months. Specific examples of outcomes include MDS-UPDRS, dopamine transporter imaging striatal uptake, vesicular monoamine transporter type-2 uptake, and serum and CSF alpha-synuclein. PD patient subsets may be defined by baseline assessments, genetic mutations, progression milestones and/or rate of clinical, imaging, or biomic change.
- Comparison between Rates of Change [ Time Frame: Study intervals ranging from 3 months to 156 months ]Comparison between the rates of change in the mean of clinical, imaging and biomic outcomes in various subsets (including, but not limited to: early PD vs. healthy subjects, PD vs. SWEDD, PD vs. prodromal, PD with and without LRRK2, GBA or SNCA mutation, unaffected LRRK2, GBA or SNCA mutation vs. healthy controls)
- Prevalence of measures of clinical, imaging and biomic outcomes in various subsets ( [ Time Frame: study intervals ranging from baseline to 156 months. ]Including, but not limited to: early PD patients, healthy subjects, PD vs SWEDDs, PD vs prodromal, PD with and without LRRK2, GBA or SNCA mutation, unaffected LRRK2, GBA or SNCA mutation carriers vs. healthy subjects.
- Predictive Value [ Time Frame: Baseline to 156 months ]To establish the predictive value of early clinical non-motor features, baseline imaging and biomic outcomes for future course of disease.
- To examine the proportion of SWEDD subjects that have a change in their clinical management at 24 months [ Time Frame: Baseline to 156 Months ]SWEDD Clinical Diagnosis and Management Questionnaire.
- Exploratory Analysis [ Time Frame: Baseline to 156 months ]Exploratory analysis to estimate the percentage of Prodromal subjects with one or more risk characteristics [hyposmia (<10th percentile for age and gender), RBD, or LRRK2, GBA or SNCA genetic mutation, and baseline DaTSCAN binding showing minimal to moderate DAT deficit] that phenoconvert to PD within 2 years, and an exploratory analysis to examine whether the baseline DaTSCAN binding or progression of clinical, imaging, or biospecimen markers may predict those subjects likely to phenoconvert.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01141023
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|Study Chair:||Kenneth L Marek, MD||Institute for Neurodegenerative Disorders|
|Study Director:||John Q. Trojanowski, MD, PhD||University of Pennsylvania|
|Study Director:||Arthur W. Toga, PhD||University of California, Los Angeles|
|Study Director:||Tatiana Froud, PhD||Indiana University|
|Study Director:||Karl Kieburtz, MD||Clinical Trials Coordination Center|
|Study Director:||Andrew Singleton, PhD||Laboratory of Neurogenetics; National Institute on Aging NIH|
|Study Director:||John P Seibyl, MD||Institute for Neurodegenerative Disorders|
|Study Director:||Christopher Coffey, PhD||Clinical Trials Statistical and Data Management Center, University of Iowa|