Systemic Synuclein Sampling Study (S4)

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02572713
• Date of registration: 07/10/2015
• Prospective Registration: No
• Primary sponsor: Michael J. Fox Foundation for Parkinson's Research
• Public title: Systemic Synuclein Sampling Study (S4) S4
• Scientific title: Systemic Synuclein Sampling Study (S4)
• Date of first enrolment: October 2015
• Target sample size: 80
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02572713
• Study type: Observational
• Phase: N/A

Countries of recruitment

Canada, United States

Contacts

• Name: Lana Chahine, MD
• Affiliation: University of Pennsylvania

Key inclusion & exclusion 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.



Age minimum: 40 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Parkinson's Disease

Intervention(s)

Drug: DaTSCAN™

Procedure: Tissue samplings

Procedure: Biofluid samplings

Primary Outcome(s)

a-syn deposits in submandibular gland [Time Frame: 24 months]

a-syn deposits in colon [Time Frame: 24 months]

a-syn levels in CSF [Time Frame: 24 months]

a-syn levels in blood [Time Frame: 24 months]

a-syn levels in saliva [Time Frame: 24 months]

a-syn deposits in skin [Time Frame: 24 months]

Secondary ID(s)

S4-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Banner Health

University of Iowa

Indiana University

Paracelsus Elena Klinik