LRRK2 and Other Novel Exosome Proteins in Parkinson's Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by University of Alabama at Birmingham
Sponsor:
Information provided by (Responsible Party):
Andrew B. West, University of Alabama at Birmingham
ClinicalTrials.gov Identifier:
NCT01860118
First received: April 19, 2013
Last updated: April 8, 2014
Last verified: April 2014
  Purpose

This proposal seeks to 1) determine whether there are biomarkers associated with Parkinson's disease (PD) susceptibility and/or progression in exosome-proteomes derived from PD patients versus controls, and 2) to determine if LRRK2 expression and/or phosphorylation are significantly lowered in the exosomes of individuals treated with the potent LRRK2 kinase inhibitor sunitinib (a multi-kinase inhibitor compound), to establish an assay for on-target effects for future LRRK2 inhibitor clinical trials.


Condition
Parkinson's Disease

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: LRRK2 and Other Novel Exosome Proteins in Parkinson's Disease

Resource links provided by NLM:


Further study details as provided by University of Alabama at Birmingham:

Primary Outcome Measures:
  • Biomarkers [ Time Frame: up to 3 years ] [ Designated as safety issue: No ]
    Biomarkers associated with Parkinson's disease (PD) susceptibility and/or progression in exosome-proteomes derived from PD patients versus controls.


Secondary Outcome Measures:
  • LRRK2 expression and/or phosphorylation [ Time Frame: up to 3 years ] [ Designated as safety issue: No ]
    Determine if LRRK2 expression and/or phosphorylation are significantly lowered in the exosomes of individuals treated with the potent LRRK2 kinase inhibitor sunitinib (a multi-kinase inhibitor compound), to establish an assay for on-target effects for future LRRK2 inhibitor clinical trials.


Biospecimen Retention:   Samples With DNA

Whole blood samples and urine


Estimated Enrollment: 700
Study Start Date: January 2013
Estimated Study Completion Date: January 2017
Estimated Primary Completion Date: April 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts
Parkinson's Disease
1) the presence of bradykinesia and either rest tremor or rigidity; 2) asymmetric onset; 3) progressive motor symptoms 4) age at onset 21-99 years.
Sunitinib
Neurologically normal patients, without history of PD, currently undergoing sunitinib treatment for cancer.
Healthy Control
Healthy controls between ages of 21-99 years and a lack of PD in first-degree blood relatives

  Eligibility

Ages Eligible for Study:   21 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population

Neurology clinic

Criteria

Inclusion Criteria:

Parkinson's disease inclusion criteria:

Inclusion criteria will be based on UK Brain Bank criteria for the clinical diagnosis of PD. These require 1) the presence of bradykinesia and either rest tremor or rigidity; 2) asymmetric onset; 3) progressive motor symptoms 4) age at onset 21-99 years.

Sunitinib cohort criteria:

Neurologically normal patients, without history of PD, currently undergoing sunitinib treatment for cancer. Urine will be collected 4-hours (peak serum drug levels in most people) after their usual AM dosage of drug (cancers include pheochromocytoma, carcinoid, or renal cancer with still normal kidney function (GFR >60), all seen at the UAB Comprehensive Cancer center). Current age from 21 to 99 will be collected.

Control inclusion criteria: ages of between 21-99 years, a lack of PD in first-degree blood relatives, and a lack of positive responses on more than 3 items on the PD Screening Questionnaire.

Exclusion Criteria:

For all subjects:

include atypical features indicative of a Parkinson-Plus disorder (Progressive Supranuclear Palsy (PSP), Multiple System Atrophy (MSA), Corticobasal Degeneration (CBD)) including cerebellar signs, supranuclear gaze palsy, apraxia and other cortical signs, or prominent autonomic failure, neuroleptic treatment at time of onset of parkinsonism, active treatment with a neuroleptic at time of study entry, History of repeated strokes with stepwise progression of parkinsonism, history of repeated head injury, history of definite encephalitis, prominent gait imbalance early in the course (< 5 years), dementia, known severe anemia (hematocrit <30), history of kidney disease and/or current or past glomerular filtration rate (GFR) <60 possibly indicative of kidney disease, or a serious comorbidity that may interfere with participation in the study.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01860118

Contacts
Contact: Andrew West, PhD 205-996-7697 abwest@uab.edu
Contact: Rachel Clark, RN 205-996-2647 rclarkrn@uab.edu

Locations
United States, Alabama
University of Alabama at Birmingham Sparks Center Recruiting
Birmingham, Alabama, United States, 35294
Contact: Andrew West, PhD    205-996-7697    abwest@uab.edu   
Contact: Rachel Clark, RN    205-996-2647    rclarkrn@uab.edu   
Principal Investigator: Andrew West, PhD         
Sponsors and Collaborators
University of Alabama at Birmingham
Investigators
Principal Investigator: Andrew West, PhD University of Alabama at Birmingham
  More Information

No publications provided

Responsible Party: Andrew B. West, Associate Professor, University of Alabama at Birmingham
ClinicalTrials.gov Identifier: NCT01860118     History of Changes
Other Study ID Numbers: 1U18NS082132
Study First Received: April 19, 2013
Last Updated: April 8, 2014
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases

ClinicalTrials.gov processed this record on August 01, 2014