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Systemic Synuclein Sampling Study (S4) (S4)

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ClinicalTrials.gov Identifier: NCT02572713
Recruitment Status : Completed
First Posted : October 9, 2015
Last Update Posted : August 17, 2017
Sponsor:
Collaborators:
Indiana University
University of Iowa
Banner Health
Paracelsus Elena Klinik
Information provided by (Responsible Party):
Lana Chahine, MD, University of Pennsylvania

Tracking Information
First Submitted Date October 7, 2015
First Posted Date October 9, 2015
Last Update Posted Date August 17, 2017
Study Start Date October 2015
Actual Primary Completion Date August 1, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 9, 2015)
  • α-syn levels in blood [ Time Frame: 24 months ]
    Blood will be analyzed using the most optimal, currently available, quantitative assays. The outcome will be expressed as a concentration of α-syn levels.
  • α-syn levels in saliva [ Time Frame: 24 months ]
    Saliva will be analyzed using the most optimal, currently available, quantitative assays. The outcome will be expressed as a concentration of α-syn levels.
  • α-syn levels in CSF [ Time Frame: 24 months ]
    CSF will be analyzed using the most optimal, currently available, quantitative assays. The outcome will be expressed as a concentration of α-syn levels.
  • α-syn deposits in skin [ Time Frame: 24 months ]
    α-syn burden in skin biopsies will be expressed as 1) simply positive or negative, i.e. whether any two slides are positive out of all examined 2) by total percentage of slides examined that are positive 3) by site of highest density of positive α-syn fibers.
  • α-syn deposits in submandibular gland [ Time Frame: 24 months ]
    α-syn burden in the submandibular tissue will be expressed as 1) simply positive or negative, i.e. whether any two slides are positive out of all examined 2) by total percentage of slides examined that are positive 3) by site of highest density of positive α-syn fibers.
  • α-syn deposits in colon [ Time Frame: 24 months ]
    α-syn burden in the colon tissue will be expressed as 1) simply positive or negative, i.e. whether any two slides are positive out of all examined 2) by total percentage of slides examined that are positive 3) by site of highest density of positive α-syn fibers.
Original Primary Outcome Measures
 (submitted: October 7, 2015)
  • Biofluid α-syn levels [ Time Frame: 24 months ]
    Whole blood, serum, plasma, saliva, and CSF α-syn will be analyzed using the most optimal, currently available, quantitative and semi-quantitative assays. The outcome will be expressed as a concentration of total α-syn and other α-syn species or a ratio of specific species to total levels.
  • α-syn deposits in skin [ Time Frame: 24 months ]
    α-syn burden in skin biopsies will be expressed as 1) simply positive or negative, i.e. whether any two slides are positive out of all examined 2) by total percentage of slides examined that are positive 3) by site of highest density of positive α-syn fibers.
  • α-syn deposits in submandibular gland [ Time Frame: 24 months ]
    α-syn burden in the submandibular tissue will be expressed as 1) simply positive or negative, i.e. whether any two slides are positive out of all examined 2) by total percentage of slides examined that are positive 3) by site of highest density of positive α-syn fibers.
  • α-syn deposits in colon [ Time Frame: 24 months ]
    α-syn burden in the colon tissue will be expressed as 1) simply positive or negative, i.e. whether any two slides are positive out of all examined 2) by total percentage of slides examined that are positive 3) by site of highest density of positive α-syn fibers.
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Systemic Synuclein Sampling Study (S4)
Official Title Systemic Synuclein Sampling Study (S4)
Brief Summary The purpose of this study is to measure alpha-synuclein in peripheral body tissues and fluids in Parkinson's disease (PD). This may help in developing better treatments for PD patients in the future.
Detailed Description This is a multi-center, cross-sectional, observational study to evaluate α-syn pathology in multiple tissues and biofluids in individual subjects with PD and HC at a single time point.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Biofluid samplings [blood, saliva, and cerebrospinal fluid (CSF)] Tissue samplings (skin, colon, submandibular gland)
Sampling Method Non-Probability Sample
Study Population Patients will be recruited through patient care clinics, physician referrals, and by reaching out to the community (e.g. PD-affiliated groups).
Condition Parkinson's Disease
Intervention
  • Procedure: Biofluid samplings
    Biofluid samplings (blood, saliva, and cerebrospinal fluid (CSF)
  • Procedure: Tissue samplings
    Tissue samplings (skin, colon, submandibular gland)
  • Drug: DaTSCAN™
    Other Name: ioflupane-123I
Study Groups/Cohorts
  • Early PD
    20 early PD not requiring dopamine replacement therapy have been enrolled.
    Interventions:
    • Procedure: Biofluid samplings
    • Procedure: Tissue samplings
    • Drug: DaTSCAN™
  • Moderate PD
    20 moderate PD on dopamine replacement therapy without motor fluctuations have been enrolled.
    Interventions:
    • Procedure: Biofluid samplings
    • Procedure: Tissue samplings
    • Drug: DaTSCAN™
  • Advanced PD
    21 advanced PD with motor fluctuations have been enrolled.
    Interventions:
    • Procedure: Biofluid samplings
    • Procedure: Tissue samplings
    • Drug: DaTSCAN™
  • Healthy Controls
    21 healthy controls have been enrolled.
    Interventions:
    • Procedure: Biofluid samplings
    • Procedure: Tissue samplings
    • Drug: DaTSCAN™
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: October 7, 2015)
80
Original Estimated Enrollment Same as current
Actual Study Completion Date August 1, 2017
Actual Primary Completion Date August 1, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria (PD subjects):

  • Male or female age 40 or older at the time of PD diagnosis.
  • Clinical diagnosis of PD based on bradykinesia plus one of the following: rest tremor or rigidity.
  • DAT deficit at screening based on visual interpretation of DaTSCAN™ imaging.
  • PD subjects will need to fall into one of the following stages:

    • Early untreated PD not requiring dopamine replacement medication (anticholinergics, MAO-B inhibitors and amantadine permitted), Hoehn and Yahr 1-2, < 2 years from diagnosis.
    • Moderate PD responsive and currently treated with dopamine replacement therapy without evidence of motor fluctuations or dyskinesias.
    • Advanced PD with motor fluctuations or dyskinesias, > 5 years from diagnosis.
  • Ability to provide written informed consent in accordance with Good Clinical Practice (GCP), International Conference on Harmonization (ICH), and local regulations.
  • Willing and able to comply with scheduled visits, required study procedures and laboratory tests.

Inclusion Criteria (HC subjects):

  • Male or female age 50 or older at the time of the screening visit
  • Ability to provide written informed consent in accordance with Good Clinical Practice (GCP), International Conference on Harmonization (ICH), and local regulations.
  • Willing and able to comply with scheduled visits, required study procedures and laboratory tests.

Exclusion Criteria (all subjects):

  • Has a history of cancer (other than basal and squamous cell skin cancers), autoimmune disorder, liver disease, or other hematological disorder within the past 5 years.
  • Current treatment with anticoagulants (e.g., Coumadin, heparin) that would preclude safe completion of the lumbar puncture (LP) and tissue biopsy procedures.
  • Current treatment with an antiplatelet agent (Plavix or aspirin >325 mg/day).
  • Has a diagnosis of diabetes mellitus requiring either an oral agent or insulin therapy.
  • A bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia.
  • Has received botulinum toxin injections to the submandibular gland within the past year.
  • Has a condition that precludes safe performance of routine LP, such as prohibitive lumbar spinal disease.
  • Has a condition that precludes the safe performance of the flexible sigmoidoscopy procedure or may interfere with obtaining evaluable colonic tissue biopsies, including a prior colonoscopy with significant findings (e.g. polyp with a positive finding, ulcerative colitis, Crohn's disease, inflammatory disease).
  • Has a condition that precludes the safe performance of the submandibular gland procedure or may interfere with obtaining evaluable submandibular tissue biopsies, including any previous or active significant disease affecting the submandibular gland (e.g. inflammatory disease, infection, tumor).
  • Has a condition that precludes the safe performance of the skin punch biopsy procedure or may interfere with obtaining evaluable skin tissue biopsies, including any previous or active significant dermatological disease (e.g. previous biopsy with any of the following findings: inflammatory disease, scar tissue, psoriasis, keloid formation, skin cancer).
  • Any other medical or psychiatric condition or laboratory abnormality, which in the opinion of the Site Investigator would preclude participation.
  • Use of investigational drugs or devices within 30 days prior to the screening visit.

Exclusion criteria (PD subjects):

  • Has other significant neurological disorders (clinically significant stroke, brain tumor, hydrocephalus, epilepsy, other neurodegenerative disorders, encephalitis, repeated head trauma, polyneuropathy).
  • Has significant autonomic dysfunction (symptomatic orthostasis, hypotension or urinary incontinence) suggestive of an atypical parkinsonism.
  • Has atypical features of parkinsonism including but not limited to supranuclear gaze palsy, early recurrent falls, corticospinal track abnormalities, cerebellar abnormalities, significant cognitive dysfunction.

Exclusion criteria (HC subjects):

  • Has a family history of PD in any first-degree relative.
  • Has a significant neurological disorder (a neurodegenerative condition, clinically significant stroke, brain tumor, hydrocephalus, epilepsy, other neurodegenerative disorders, encephalitis, repeated head trauma, polyneuropathy).
  • Has a Montreal Cognitive Assessment (MoCA) score of less than 26.
  • Has a diagnosis of REM sleep behavior disorder.
  • Has a primary dystonia, restless legs syndrome, essential tremor, or other movement disorder.
Sex/Gender
Sexes Eligible for Study: All
Ages 40 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers Yes
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02572713
Other Study ID Numbers S4-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Lana Chahine, MD, University of Pennsylvania
Study Sponsor Michael J. Fox Foundation for Parkinson's Research
Collaborators
  • Indiana University
  • University of Iowa
  • Banner Health
  • Paracelsus Elena Klinik
Investigators
Principal Investigator: Lana Chahine, MD University of Pennsylvania
PRS Account Michael J. Fox Foundation for Parkinson's Research
Verification Date August 2017