CA SE STUDY NO. 3
S4 Study Site Selection
Background
The Systemic Synuclein Sampling Study (S4) is a multicenter,
cross-sectional, observational study sponsored by The Michael
J. Fox Foundation. The primary objective of the study is to
better understand the progression of Parkinson's disease (PD)
by identifying the optimal biofluids and tissues for measuring
the protein alpha-synuclein outside of the brain as a potential
biomarker in individuals with PD. The secondary objective of
the study is to create standard operating procedures for the
collection and assessment of multiple tissues and biofluids to
better understand alpha-synuclein’s potential as a biomarker for
PD. (To learn more about S4, visit michaeljfox.org/s4.)
Study Design
Participants
S4 sought to enroll 80 participants meeting the following
characteristics [Table 1]:
Table 1. Target Enrollment, S4 Study
Target
Enrollment
(n =)
Participant
Characteristics
20 Individuals with early PD not
requiring dopamine replacement
therapy
20 Individuals with moderate PD on
dopamine replacement therapy
without motor fluctuations
20
20
Individuals with advanced PD with
motor fluctuations
Controls
*https://www.futuremedicine.com/doi/10.2217/bmm-2016-0366
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Accelerating Clinical Trials: Best Practices for Recruitment and Retention
Procedures
The section below provides a condensed overview of the
procedures that study participants underwent as part of S4. For
additional information on the procedures and scientific rationale
used in S4, please refer to the study report.*
+ + Screening Visit
– – Study staff informed and consented study participants
for participation (i.e., study purpose, procedures,
potential risks and benefits were explained, and consent
to participate was obtained). Their medical and
family histories and any medications were reviewed.
Participants’ vital signs were taken, and a general physical
and neurological exam were performed. All participants
underwent MDS-UPDRS, Hoehn and Yahr, MoCA,
SCOPA-AUT and UPSIT exams. Individuals with PD
received Modified Schwab & England Activities of
Daily Living Scale and PD stage assignment.
+ + Biofluid Collection and Skin Biopsy Visit
– – Whole blood, serum and plasma were collected through
routine venipuncture.
– – Cerebrospinal fluid was collected through lumbar
puncture under local anesthesia.
– – Saliva was collected through 20-minute active
drool collection.
– – Approximately four samples of thigh and upper
back/shoulder skin were biopsied using skin punch
under local anesthesia.
+ + Colon Biopsy Visit
– – Approximately eight samples of colonic tissue
were biopsied from study participants using routine
clinical procedures.
+ + Submandibular Gland Biopsy Visit
– – Approximately five samples of submandibular (type of
salivary) gland tissue were biopsied using a 16-gauge
needle inserted through the neck under local anesthesia.