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NCT04477785RecruitingAccepts Healthy Volunteers

PPMI Clinical - Establishing a Deeply Phenotyped PD Cohort

Michael J. Fox Foundation for Parkinson's Research

Start Date

7/1/2020

Completion Date

12/1/2033

Summary

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.

Detailed Description

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.

Eligibility Criteria

Age Range: 30 years to No maximum

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.

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Conditions

Parkinson Disease

Locations

University of Alabama at Birmingham

Birmingham, Alabama 35294

United States

Barrow Neurological Institute

Phoenix, Arizona 85013

United States

Mayo Foundation for Medical Education and Research

Scottsdale, Arizona 85259

United States

Banner Research Institute

Sun City, Arizona 85351

United States

University of California San Diego

La Jolla, California 92093-0948

United States

Keck School of Medicine of USC

Los Angeles, California 90033

United States

University of California, San Francisco

San Francisco, California 94115

United States

University of Colorado Anschutz Medical Campus

Aurora, Colorado 80045

United States

Institute For Neurodegenerative Disorders

New Haven, Connecticut 06510

United States

Parkinson's Disease& Movement Disorder Center of Boca Raton

Boca Raton, Florida 33486

United States

University of Florida

Gainesville, Florida 32608

United States

University of South Florida

Tampa, Florida 33606

United States

Emory University School of Medicine

Atlanta, Georgia 30329

United States

Northwestern University

Chicago, Illinois 60611

United States

University of Kansas Medical Center

Kansas City, Kansas 66160

United States

Johns Hopkins University

Baltimore, Maryland 21287

United States

Boston University

Boston, Massachusetts 02118

United States

Massachusetts General Hospital

Boston, Massachusetts 02446

United States

University of Michigan

Ann Arbor, Michigan 48109

United States

Cleveland Clinic Lou Ruvo Center for Brain Health

Las Vegas, Nevada 89106

United States

Beth Israel Medical Center

New York, New York 10003

United States

NYU Langone Health

New York, New York 10017

United States

University of Rochester

Rochester, New York 14620

United States

University of Cincinnati/Cincinnati Children's Hospital

Cincinnati, Ohio 45219

United States

Cleveland Clinic

Cleveland, Ohio 44195

United States

Oregon Health &Science University

Portland, Oregon 97239

United States

University of Pennsylvania

Philadelphia, Pennsylvania 19107

United States

University of Pittsburgh

Pittsburgh, Pennsylvania 15213

United States

Baylor College of Medicine

Houston, Texas 77030

United States

Univ of Washington and VA Puget Sound Health Care System

Seattle, Washington 98104

United States

Innsbruck Medical University

Innsbruck, 6020

Austria

The Ottawa Hospital - Civic Campus

Ottawa, Ontario K1Y 4E9

Canada

Toronto Western Hospital

Toronto, Ontario M5T 2S8

Canada

McGill University

Montreal, Quebec H3A2B4

Canada

Philipps-University of Marburg

Hessen, 35043

Germany

Paracelsus-Elena Klinik

Kassel, 34128

Germany

University of Luebeck

Lübeck, 23562

Germany

University of Tuebingen

Tübingen, 72076

Germany

Foundation for Biomedical Research of the Academy of Athens

Athens, Athens 11523

Greece

Tel Aviv Sourasky Medical Center

Tel Aviv, Tel Aviv 64239

Israel

University of Salerno

Salerno, Salerno 84131

Italy

Parkinson Research Clinic

Luxembourg, L-1257

Luxembourg

Radboud University

Nijmegen, Gelderland 6525 GC

Netherlands

Lagos College of Medicine, University of Lagos

Lagos, Lagos 121010

Nigeria

Hospital Clinic de Barcelona

Barcelona, Barcelona 08036

Spain

Hospital Donostia

Donostia / San Sebastian, San Sebastian 20014

Spain

Queen Mary University of London

London, Britain EC1M 6BQ

United Kingdom

Newcastle University

Newcastle upon Tyne, Tyne and Wear NE45PL

United Kingdom

Imperial College London

London, W12 0NN

United Kingdom

John Radcliffe Hospital Oxford and Oxford University

Oxford, Oxford, OX3 9DU

United Kingdom