Michael J. Fox Foundation for Parkinson's Research4,500 enrolled
Overview
The Parkinson Progression Marker Initiative (PPMI) is a longitudinal, observational, multi-center natural history study to assess progression of clinical features, digital outcomes, and imaging, biologic and genetic markers of Parkinson's disease (PD) progression in study participants with manifest PD, prodromal PD, and healthy controls.
The overall goal of PPMI is to identify markers of disease progression for use in clinical trials of therapies to reduce progression of PD disability.
PPMI is a broad program, expanding the goals of the original PPMI study, that includes this PPMI Clinical protocol, as well as other program initiatives such as the PPMI Remote, PPMI Digital App and PPMI Online protocols. Participants in PPMI may be asked to be enrolled in other PPMI program protocols, but depending on their method of recruitment, participants may be enrolled sequentially in varying order, as appropriate. PPMI participants may also be asked to participate in additional PPMI program initiatives (as they are developed), which may only involve a subset of PPMI participants based on their cohort designation and/or site location.
7.1 Healthy Controls (HC) Note: Active Healthy controls previously enrolled in PPMI do not require re-assessment of eligibility criteria listed below for enrollment in PPMI Clinical. Active participants do need to be able to provide informed consent for PPMI Clinical participation (includes use of a designated research proxy).
7.1.1 Inclusion Criteria (HC)
1. Male or female age 57 years or older at Screening visit.
2. Individuals taking any of the following drugs: alpha methyldopa, methylphenidate, amphetamine derivatives or modafinil, must be willing and medically able to hold the medication for at least 5 half-lives before SPECT imaging.
3. Confirmation that participant is eligible based on Screening SPECT imaging.
4. Able to provide informed consent.
5. Either is male, or is female and meets additional criteria below, as applicable:
* Female of childbearing potential who is not pregnant, lactating, or planning pregnancy during the study and has a negative pregnancy test on day of Screening SPECT imaging test prior to injection of DaTscanTM.
7.1.2 Exclusion Criteria (HC)
1. First degree relative with PD (i.e., biologic parent, sibling, child).
2. Current or active clinically significant neurological disorder (in the opinion of the Investigator).
3. Previously obtained MRI scan with evidence of clinically significant neurological disorder (in the opinion of the Investigator).
4. Received any of the following drugs: dopamine receptor blockers (neuroleptics), metoclopramide and reserpine within 6 months of Screening visit.
5. Current treatment with anticoagulants (e.g., coumadin, heparin, oral thrombin inhibitors) that might preclude safe completion of the lumbar puncture.
6. Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia.
7. Any other medical or psychiatric condition or lab abnormality, which in the opinion of the investigator might preclude participation.
8. Any other reason that, in the opinion of the investigator, would render the participant unsuitable for study enrollment.
7.2 Parkinson's Disease (PD) Note: Active PD participants previously enrolled in PPMI do not require re-assessment of eligibility criteria listed below for enrollment in PPMI Clinical. Active participants do need to be able to provide informed consent for PPMI Clinical participation (includes use of a designated research proxy).
7.2.1 Inclusion Criteria (PD)
1. Male or female age 30 years or older at Screening Visit.
2. A diagnosis of Parkinson's disease for 2 years or less at Screening Visit.
3. Not expected to require PD medication within at least 6 months from Baseline.
4. Patients must have at least two of the following: resting tremor, bradykinesia, rigidity (must have either resting tremor or bradykinesia); OR either asymmetric resting tremor or asymmetric bradykinesia.
5. Hoehn and Yahr stage I or II at Baseline.
6. Individuals taking any of the following drugs: alpha methyldopa, methylphenidate, amphetamine derivatives or modafinil, must be willing and medically able to hold the medication for at least 5 half-lives before SPECT imaging.
7. Confirmation that participant is eligible based on Screening SPECT imaging.
8. Able to provide informed consent.
9. Either is male, or is female and meets additional criteria below, as applicable:
* Female of childbearing potential who is not pregnant, lactating, or planning pregnancy during the study and has a negative pregnancy test on day of Screening SPECT imaging test prior to injection of DaTscanTM.
7.2.2 Exclusion Criteria (PD)
1. Currently taking levodopa, dopamine agonists, MAO-B inhibitors, amantadine or another PD medication, except for low-dose treatment of restless leg syndrome (with permission of medical monitor).
2. Has taken levodopa, dopamine agonists, MAO-B inhibitors or amantadine within 60 days of Baseline visit.
3. Has taken levodopa or dopamine agonists prior to Baseline visit for more than a total of 90 days.
4. Atypical PD syndromes due to either drugs (e.g., metoclopramide, flunarizine, neuroleptics) or metabolic disorders (e.g., Wilson's disease), encephalitis, or degenerative diseases (e.g., progressive supranuclear palsy).
5. A clinical diagnosis of dementia as determined by the investigator.
6. Previously obtained MRI scan with evidence of clinically significant neurological disorder (in the opinion of the Investigator).
7. Received any of the following drugs: dopamine receptor blockers (neuroleptics), metoclopramide and reserpine within 6 months of Screening visit.
8. Current treatment with anticoagulants (e.g., coumadin, heparin, oral thrombin inhibitors) that might preclude safe completion of the lumbar puncture.
9. Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia.
10. Any other medical or psychiatric condition or lab abnormality, which in the opinion of the investigator might preclude participation.
11. Any other reason that, in the opinion of the investigator, would render the participant unsuitable for study enrollment.
7.3 Parkinson's Disease (PD) with LRRK2 or GBA variant Note: Active PD participants previously enrolled in PPMI do not require re-assessment of eligibility criteria listed below for enrollment in PPMI Clinical. Active participants do need to be able to provide informed consent for PPMI Clinical participation (includes use of a designated research proxy).
7.3.1 Inclusion Criteria (PD ¬- LRRK2 or GBA)
1. Male or female age 30 years or older at Screening Visit.
2. A diagnosis of Parkinson's disease for 2 years or less at Screening Visit.
3. Patients must have at least two of the following: resting tremor, bradykinesia, rigidity (must have either resting tremor or bradykinesia); OR either asymmetric resting tremor or asymmetric bradykinesia.
4. Hoehn and Yahr stage I or II at Baseline.
5. Confirmation of causative LRRK2 or GBA (willingness to undergo genetic testing as part of genetic screening and be informed of genetic testing results, or approved documentation of prior genetic testing results).
6. Individuals taking any of the following drugs: alpha methyldopa, methylphenidate, amphetamine derivatives or modafinil, must be willing and medically able to hold the medication for at least 5 half-lives before SPECT imaging.
7. Confirmation that participant is eligible based on Screening SPECT imaging.
8. Able to provide informed consent.
9. Either is male, or is female and meets additional criteria below, as applicable:
* Female of childbearing potential who is not pregnant, lactating, or planning pregnancy during the study and has a negative pregnancy test on day of Screening SPECT imaging test prior to injection of DaTscanTM.
7.3.2 Exclusion Criteria (PD - LRRK2 or GBA)
1. Received any of the following drugs: dopamine receptor blockers (neuroleptics), metoclopramide and reserpine within 6 months of Screening visit.
2. Current treatment with anticoagulants (e.g., coumadin, heparin) that might preclude safe completion of the lumbar puncture.
3. Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia.
4. Any other medical or psychiatric condition or lab abnormality, which in the opinion of the investigator might preclude participation.
5. Any other reason that, in the opinion of the investigator, would render the participant unsuitable for study enrollment.
7.4 Parkinson's Disease (PD) with SNCA or rare genetic variant Note: Active PD participants previously enrolled in PPMI do not require re-assessment of eligibility criteria listed below for enrollment in PPMI clinical. Active participants do need to be able to provide informed consent for PPMI Clinical participation (includes use of a designated research proxy).
7.4.1 Inclusion Criteria (PD - SNCA or rare genetic variant (such as Parkin or Pink1))
1. Male or female age 30 years or older at Screening Visit.
2. Parkinson's disease diagnosis at Screening Visit.
3. Patients must have at least two of the following: resting tremor, bradykinesia, rigidity (must have either resting tremor or bradykinesia); OR either asymmetric resting tremor or asymmetric bradykinesia.
4. Hoehn and Yahr stage I, II, or III at Baseline.
5. Confirmation of causative SNCA or rare genetic variant (such as Parkin or Pink1) (willingness to undergo genetic testing as part of genetic screening and be informed of genetic testing results, or approved documentation of prior genetic testing results).
6. Individuals taking any of the following drugs: alpha methyldopa, methylphenidate, amphetamine derivatives or modafinil, must be willing and medically able to hold the medication for at least 5 half-lives before SPECT imaging.
7. Confirmation that participant is eligible based on Screening SPECT imaging.
8. Able to provide informed consent.
9. Either is male, or is female and meets additional criteria below, as applicable:
* Female of childbearing potential who is not pregnant, lactating, or planning pregnancy during the study and has a negative pregnancy test on day of Screening SPECT imaging test prior to injection of DaTscanTM.
7.4.2 Exclusion Criteria (PD - SNCA or rare genetic variant (such as Parkin or Pink1))
1. Received any of the following drugs: dopamine receptor blockers (neuroleptics), metoclopramide and reserpine within 6 months of Screening visit.
2. Current treatment with anticoagulants (e.g., coumadin, heparin) that might preclude safe completion of the lumbar puncture.
3. Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia.
4. Any other medical or psychiatric condition or lab abnormality, which in the opinion of the investigator might preclude participation.
5. Any other reason that, in the opinion of the investigator, would render the participant unsuitable for study enrollment.
7.5 Prodromal Note: Active Prodromal participants previously enrolled in PPMI do not require re-assessment of eligibility criteria listed below for enrollment in PPMI Clinical. Active participants do need to be able to provide informed consent for PPMI Clinical participation (includes use of a designated research proxy).
The specific predictive eligibility criteria for participants recruited through PPMI Remote to advance to PPMI Clinical will be iteratively optimized based on data collected from these studies.
7.5.1 Inclusion criteria (Prodromal)
For Screening:
1. Confirmation that participant is eligible based on centrally determined predictive criteria including the University of Pennsylvania Smell Identification Test (UPSIT).
* For participants in PPMI Remote, referral to the clinical site confirms predictive eligibility.
* For participants identified by the clinical site, predictive criteria are based on generalized risk such as first degree biologic relative, known risk of PD including RBD, or known genetic variants associated with PD risk.
Additionally, confirmation of UPSIT eligibility during the Screening visit prior to SPECT Imaging.
2. Male or female age 60 years or older (except age 30 years or older for SNCA, or rare genetic variants (such as Parkin or Pink1) participants).
3. Individuals taking any of the following drugs: alpha methyldopa, methylphenidate, amphetamine derivatives or modafinil, must be willing and medically able to hold the medication for at least 5 half-lives before SPECT imaging.
4. Able to provide informed consent.
5. Either is male, or is female and meets additional criteria below, as applicable:
• Female of childbearing potential who is not pregnant, lactating, or planning pregnancy during the study and has a negative pregnancy test on day of Screening SPECT imaging test prior to injection of DaTscanTM.
For continuation to Baseline visit and ongoing follow-up:
6. Confirmation that participant is eligible based on \*Screening SPECT imaging.
* Screening SPECT Imaging eligibility:
Based on the results of the SPECT imaging test, Prodromal participants eligible to continue their participation in PPMI Clinical will be asked to return for their PPMI Clinical baseline visit. Neither the participant nor the site investigator will be made aware of the participant's DAT status during the study.
* It is anticipated that approximately 6,000 participants will complete a screening visit to undergo DAT imaging. Approximately 2,000 participants will be eligible to continue their participation in PPMI Clinical (those not eligible to proceed will remain in PPMI Remote, as applicable).
* All participants with DAT deficit will be eligible to continue their participation in PPMI Clinical. It is estimated that about 75% of eligible participants will have a DAT deficit (defined by a hybrid of visual assessment and quantitative striatal specific binding analysis).
* Some participants without DAT deficit will also be eligible to continue their participation in PPMI Clinical. These participants will be chosen based on DAT binding that is reduced from age expected but it not outside the normal range and/or from individuals with high-risk of PD including RBD, LRRK2, GBA, SNCA, or rare genetic variants (such as Parkin or Pink1) that do not demonstrate DAT deficit. It is estimated that about 25% of eligible participants will not have a DAT deficit.
* It is anticipated that approximately 30% of the PPMI Clinical prodromal participants with DAT deficit will phenoconvert to motor parkinsonism during a 3 to 5-year follow-up.
7.5.2 Exclusion Criteria (Prodromal)
1. Clinical diagnosis of PD at screening, other parkinsonism, or dementia.
2. Received any of the following drugs: dopamine receptor blockers (neuroleptics), metoclopramide and reserpine within 6 months of Baseline Visit.
3. Current treatment with anticoagulants (e.g. coumadin, heparin) that might preclude safe completion of the lumbar puncture.
4. Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia.
5. Any other medical or psychiatric condition or lab abnormality, which in the opinion of the investigator might preclude participation.
6. Currently taking levodopa, dopamine agonists, MAO-B inhibitors, amantadine or another PD medication, except for low-dose treatment of restless leg syndrome (with permission of medical monitor).
7. Has taken levodopa, dopamine agonists, MAO-B inhibitors or amantadine within 60 days of Baseline visit. except for low-dose treatment of restless leg syndrome (with permission of medical monitor).
8. Any other reason that, in the opinion of the investigator, would render the participant unsuitable for study enrollment.
Locations (50)
Outcomes
Primary Outcomes
Establish standardized protocols for acquisition, transfer and analysis of clinical, digital, imaging, biologic and genetic data that can be used by the PD research community.
This protocol will build on the existing PPMI infrastructure
Time frame: Baseline to 156 months
Comprehensive and uniformly acquired dataset
Develop a comprehensive and uniformly acquired clinical, digital and imaging dataset and repository of biological and genetic samples that would be available to the PD research community to test hypotheses of the underlying molecular pathobiology of PD, enable modeling of PD progression to identify clinical and/or data driven PD progression sub-sets, and inform studies testing PD therapeutics (for examples, clinical trials targeting synuclein, LRRK2, GBA as well as other targets)
Time frame: Baseline to 156 months
Comparison between Rates of Change
Use clinical and biological data to estimate the mean rates of change and the variability around the mean of clinical, digital, imaging, biological and genetic outcomes in study participants with PD diagnosis (including patients with a LRRK2, GBA, SNCA or rare genetic variants (such as Parkin or Pink1) and individuals with prodromal Parkinson's disease (including individuals with REM sleep behavior disorder (RBD)), olfactory loss, LRRK2, GBA, SNCA or rare genetic variants (such as Parkin or Pink1) and/or other risk factors for PD with and without dopamine transporter (DAT) deficit and in healthy participants.
Time frame: Study intervals ranging from 3 months to 156 months
Prevalence of measures of clinical, imaging and biomic outcomes in various subsets
Confirm existing and identify novel clinical, digital, imaging, biologic and genetic PD progression markers to identify quantitative individual measures or combinations of measures that demonstrate optimum interval change in study participants with PD diagnosis (including patients with a LRRK2, GBA, SNCA or rare genetic variants (such as Parkin or Pink1)) and individuals with prodromal Parkinson's disease (including individuals with RBD, olfactory loss, a LRRK2, GBA, SNCA or rare genetic variants (such as Parkin or Pink1) and/or other risk factors for PD with and without DAT deficit in comparison to healthy controls or in sub-sets of study participants with PD diagnosis or prodromal PD defined by baseline assessments, progression milestones and/or rate of clinical, digital, imaging, biologic and genetic change, or other measures.
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University of Alabama at Birmingham
Birmingham, Alabama, United States
RECRUITING
Barrow Neurological Institute
Phoenix, Arizona, United States
RECRUITING
Mayo Foundation for Medical Education and Research
Scottsdale, Arizona, United States
RECRUITING
Banner Research Institute
Sun City, Arizona, United States
RECRUITING
University of California San Diego
La Jolla, California, United States
RECRUITING
Keck School of Medicine of USC
Los Angeles, California, United States
RECRUITING
University of California, San Francisco
San Francisco, California, United States
RECRUITING
University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States
RECRUITING
Institute For Neurodegenerative Disorders
New Haven, Connecticut, United States
RECRUITING
Parkinson's Disease& Movement Disorder Center of Boca Raton
Boca Raton, Florida, United States
RECRUITING
...and 40 more locations
Time frame: study intervals ranging from baseline to 156 months.
Establish the probability of phenoconversion to PD
Evaluate the probability of phenoconversion to PD for individuals with prodromal PD enrolled in the prodromal cohorts (including individuals with RBD, olfactory loss, a LRRK2, GBA, SNCA or rare genetic variants (such as Parkin or Pink1) and/ or other risk factors for PD with and without DAT deficit).
Time frame: study intervals ranging from baseline to 156 months.