Clinical trial NCT03545425: Fox BioNet Project: LRRK2-002
Official title: FoxBioNet: LRRK2-002: Detection of LRRK2 Activity in Human Biospecimens
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Parkinson Disease
Keywords: Parkinson's Disease
Biomarker
LRRK2
Lead sponsor: Michael J. Fox Foundation for Parkinson's Research
Sponsor class: OTHER
Collaborators: name: University Health Network, Toronto
name: Indiana University
name: University of Rochester
First posted: 2018-06-04
Enrollment: 114
Sex: ALL
Minimum age (years): 30
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 30 LRRK2 G2019S Manifesting carriers, 30 Idiopathic PD subjects, 30 LRRK2 G2019S Non-Manifesting Carriers, 30 Healthy Controls

Brief summary: The overall objective of this study is to determine whether LRRK2 kinase activity and/or mitochondrial DNA (mtDNA) damage could serve as potential biomarkers in PD.

Detailed description: Primary Objectives:

* Assess the levels of phosphorylated LRRK2 and LRRK2-phosphorylated Rabs, as measures of LRRK2 kinase activity, in PBMCs and neutrophils from LRRK2 PD, idiopathic PD, non-manifesting LRRK2 mutation carriers and healthy controls.
* Assess the levels of mtDNA damage in buffy coat from LRRK2 PD, idiopathic PD, non-manifesting LRRK2 mutation carriers and healthy controls.
* Correlate LRRK2 kinase activity to mtDNA damage in blood from LRRK2 PD, idiopathic-PD, non-manifesting LRRK2 mutation carriers and healthy controls.

Secondary Objectives:

* To assess the ability of the network to efficiently conduct a study involving biosample collection for PD research. Efficiency will be assessed using measures of the time taken to meet specific milestones within the study.
* To assess the ability of the network to collect high quality biospecimens adhering to agreed-upon, standardized protocols
* To gauge the willingness of participants to participate in subsequent Fox BioNet studies

Eligibility criteria: Inclusion Criteria:

LRRK2 Parkinson Disease (PD) Subjects:

* Patients must have confirmed LRRK2 mutation
* Patients must meet the MDS criteria for Parkinson's disease
* Disease duration: any
* Male or female age 30 years or older at time of PD diagnosis.

Idiopathic PD Subjects:

* Patients must meet the MDS criteria for Parkinson's disease.
* Disease duration: any
* Male or female age 30 years or older at time of PD diagnosis.

Non-manifesting LRRK2 mutation carriers:

* Patients must have confirmed LRRK2 mutation
* Male or female age 30 years or older at Screening.

Control (C) Subjects:

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

Exclusion Criteria:

LRRK2 Parkinson Disease (PD) Subjects:

* Inability to provide informed consent
* Participation in a blinded clinical trial of any kind or an unblinded trial of an investigational product that is not currently approved for use in humans.
* Treatment for cancer in the last 5 years.

Idiopathic PD Subjects:

* Inability to provide informed consent
* Participation in a blinded clinical trial of any kind or an unblinded trial of an investigational product that is not currently approved for use in humans.
* Treatment for cancer in the last 5 years.

Non-manifesting LRRK2 mutation carriers:

* Inability to provide informed consent
* Participation in a blinded clinical trial of any kind or an unblinded trial of an investigational product that is not currently approved for use in humans.
* Treatment for cancer in the last 5 years.

Control Subjects:

* Inability to provide informed consent
* Participation in a blinded clinical trial of any kind or an unblinded trial of an investigational product that is not currently approved for use in humans.
* Treatment for cancer in the last 5 years

Primary outcomes:
measure: Phosphorylated LRRK2 and LRRK2-phosphorylated Rabs
timeFrame: 7 months
measure: mtDNA damage in buffy coat
timeFrame: 7 months
measure: Correlate LRRK2 kinase activity to mtDNA damage
timeFrame: 7 months
Secondary outcomes:
measure: Assess the ability of the network to efficiently conduct a study
timeFrame: 7 months
measure: Assess the ability of the network to collect high quality biospecimens
timeFrame: 7 months
measure: To gauge the willingness of participants to participate in subsequent Fox BioNet studies
timeFrame: 7 Months
Other outcomes:
Locations:
city: Boca Raton
country: United States
facility: Charles E. Schmidt College of Medicine, Florida Atlantic University
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Portland
country: United States
facility: Oregon Health and Sciences University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
Study officials:
affiliation: University Health Network, Toronto
name: Connie Marras, MD
role: PRINCIPAL_INVESTIGATOR
References: