PPMI Clinical - Establishing a Deeply Phenotyped PD Cohort

Recruiting

• Conditions: Parkinson Disease

• Registration Number: NCT04477785
• Lead Sponsor: Michael J. Fox Foundation for Parkinson's Research

Brief 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.

Study Timeline

• Study Start: 2020-07-01
• Study First Submitted: 2020-07-15
• Study First Submitted QC: 2020-07-15
• Study First Posted: 2020-07-20
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-15
• Last Update Posted: 2025-07-16
• Primary Completion: 2033-12
• Study Completion: 2033-12-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 4500

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Comparison between Rates of Change	Study intervals ranging from 3 months to 156 months	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.
Establish the probability of phenoconversion to PD	study intervals ranging from baseline to 156 months.	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).
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.	Baseline to 156 months	This protocol will build on the existing PPMI infrastructure
Comprehensive and uniformly acquired dataset	Baseline to 156 months	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)
Prevalence of measures of clinical, imaging and biomic outcomes in various subsets	study intervals ranging from baseline to 156 months.	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.

Trial Locations

Locations (50)

University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

Barrow Neurological Institute; 🇺🇸 Phoenix, Arizona, United States

Mayo Foundation for Medical Education and Research; 🇺🇸 Scottsdale, Arizona, United States

Banner Research Institute; 🇺🇸 Sun City, Arizona, United States

University of California San Diego; 🇺🇸 La Jolla, California, United States

Keck School of Medicine of USC; 🇺🇸 Los Angeles, California, United States

University of California, San Francisco; 🇺🇸 San Francisco, California, United States

University of Colorado Anschutz Medical Campus; 🇺🇸 Aurora, Colorado, United States

Institute For Neurodegenerative Disorders; 🇺🇸 New Haven, Connecticut, United States

Parkinson's Disease& Movement Disorder Center of Boca Raton; 🇺🇸 Boca Raton, Florida, United States

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