Scottsdale, Arizona 85259

  • Parkinson's Disease

Purpose:

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.


Study summary:

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.


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.


Study is Available At:


Original ID:

S4-001


NCT ID:

NCT02572713


Secondary ID:


Study Acronym:

S4


Brief Title:

Systemic Synuclein Sampling Study (S4)


Official Title:

Systemic Synuclein Sampling Study (S4)


Overall Status:

Completed


Study Phase:

N/A


Genders:

N/A


Minimum Age:

40 Years


Maximum Age:

N/A


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

Michael J. Fox Foundation for Parkinson's Research


Oversight Authority:

United States: Institutional Review Board


Reasons Why Stopped:


Study Type:

Observational


Study Design:


Number of Arms:

0


Number of Groups:

4


Total Enrollment:

80


Enrollment Type:

Actual


Overall Contact Information

Official Name:Lana Chahine, MD
Principal Investigator
University of Pennsylvania

Study Dates

Start Date:October 2015
Completion Date:August 1, 2017
Completion Type:Actual
Primary Completion Date:August 1, 2017
Primary Completion Type:Actual
Verification Date:August 2017
Last Changed Date:August 14, 2017
First Received Date:October 7, 2015

Study Outcomes

Outcome Type:Primary Outcome
Measure:α-syn deposits in colon
Time Frame:24 months
Safety Issues:False
Description: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
Outcome Type:Primary Outcome
Measure:α-syn deposits in submandibular gland
Time Frame:24 months
Safety Issues:False
Description: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 positiv
Outcome Type:Primary Outcome
Measure:α-syn deposits in skin
Time Frame:24 months
Safety Issues:False
Description: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 fib
Outcome Type:Primary Outcome
Measure:α-syn levels in CSF
Time Frame:24 months
Safety Issues:False
Description:CSF will be analyzed using the most optimal, currently available, quantitative assays. The outcome will be expressed as a concentration of α-syn levels.
Outcome Type:Primary Outcome
Measure:α-syn levels in saliva
Time Frame:24 months
Safety Issues:False
Description:Saliva will be analyzed using the most optimal, currently available, quantitative assays. The outcome will be expressed as a concentration of α-syn levels.
Outcome Type:Primary Outcome
Measure:α-syn levels in blood
Time Frame:24 months
Safety Issues:False
Description:Blood will be analyzed using the most optimal, currently available, quantitative assays. The outcome will be expressed as a concentration of α-syn levels.

Study Interventions

Intervention Type:Procedure
Name:Biofluid samplings
Description:Biofluid samplings (blood, saliva, and cerebrospinal fluid (CSF)
Arm Name:Early PD
Intervention Type:Procedure
Name:Tissue samplings
Description:Tissue samplings (skin, colon, submandibular gland)
Arm Name:Early PD
Intervention Type:Drug
Name:DaTSCAN™
Arm Name:Early PD
Other Name:ioflupane-123I

Study Arms

Study Arm Type:Other
Arm Name:Healthy Controls
Description:21 healthy controls have been enrolled.
Study Arm Type:Other
Arm Name:Advanced PD
Description:21 advanced PD with motor fluctuations have been enrolled.
Study Arm Type:Other
Arm Name:Moderate PD
Description:20 moderate PD on dopamine replacement therapy without motor fluctuations have been enrolled.
Study Arm Type:Other
Arm Name:Early PD
Description:20 early PD not requiring dopamine replacement therapy have been enrolled.

Study Agencies

Agency Class:Other
Agency Type:Lead Sponsor
Agency Name:Michael J. Fox Foundation for Parkinson's Research
Agency Class:Other
Agency Type:Collaborator
Agency Name:Indiana University

Samples and Retentions

Sample Retention:Samples With DNA
Description: Biofluid samplings [blood, saliva, and cerebrospinal fluid (CSF)] Tissue samplings (skin, colon, submandibular gland)
Study Population: Patients will be recruited through patient care clinics, physician referrals, and by reaching out to the community (e.g. PD-affiliated groups).
Sample Method:Non-Probability Sample

Study References

There are no available Study References

Data Source: ClinicalTrials.gov

Date Processed: March 30, 2020

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