Primary objective: To evaluate the role of existing and novel quantitative biomarkers in understanding the heterogeneity in the Parkinson*s phenotype (i.e. rate of progression, cognitive and neuropsychiatric impairment and motor dysfunction) and…
ID
Source
Brief title
Condition
- Movement disorders (incl parkinsonism)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
main study parameters are biomarker concentrations (i.e. blood, CSF & feces),
skin alpha-synuclein (α-syn) aggregation, ADR occurrence and cognition scores.
Secondary outcome
nvt
Background summary
Pharmacologic treatment of Parkinson*s disease (PD) is mainly aiming to
alleviate motor or neuropsychiatric symptoms and does not alter disease
progression. Treatment follows a *one-size-fits-all* approach and does not
consider genetic factors underlying between-patient differences in treatment
response and susceptibility to adverse drug reactions (ADRs).
Study objective
Primary objective: To evaluate the role of existing and novel quantitative
biomarkers in understanding the heterogeneity in the Parkinson*s phenotype
(i.e. rate of progression, cognitive and neuropsychiatric impairment and motor
dysfunction) and predicting treatment response as well as the occurrence of
ADRs over a three-year period.
Secondary Objective: To develop a biobank containing comprehensive and
uniformly acquired longitudinal clinical data and biological samples for
identification and validation of biomarker panels and data-driven approaches
to unravel heterogeneity in the Parkinson*s phenotype (i.e. rate of
progression, cognitive and neuropsychiatric impairment and motor dysfunction),
treatment response and occurrence of ADRs.
Study design
longitudinal cohort study
Study burden and risks
Participants are invited to come to the study site in Leiden (LUMC), Amsterdam
(AMC/VU), Rotterdam (Erasmus), Utrecht/Woerden (Antonius) or Amersfoort
(Meander) for 2-3,5 hours of data collection four times during the 3-year study
duration and coupled to this visit perform an at home assessment (i.e.
questionnaires (115-160 minutes), phone interview (10-50min) and wearables (1
week) and optionally a smartphone application (1 week)). Controls will be
assessed twice. All study assessments are routine exams done in standard
clinical practice and are generally well tolerated. Blood, feces (all visits),
CSF (only at first and third visit in 100 patients and 50 controls only), MRI
(only at first visit in 100 patients) and skin biopsies (only at first and
third visit in 400 patients and 100 controls only) will be collected. Blood
draws, lumbar punctures and skin biopsies come with a small discomfort. Risks
associated with venous blood punctures, lumbar punctures and skin biopsies
include a local hematoma and, rarely, an infection. These risks will be
minimized by the applied puncture procedures carried out by experienced
physicians/nurses.
Wrist and back sensors will be worn daily for up to 24 hours, once a year for a
one-week period each in patients and twice in controls. These small,
unobtrusive electronic devices are easily applied and poses no significant
safety issues. During this week, participant will perform daily active tests of
max. 30 min a day. In addition they will fill-in daily questionnaires with a
duration of max. 5 min a day. Optionally, a smartphone application will be
used to passively collect data and 1 additional test is added to the daily
active tests.
De Boelelaan 1117
Amsterdam 1081 HV
NL
De Boelelaan 1117
Amsterdam 1081 HV
NL
Listed location countries
Age
Inclusion criteria
Prior to enrollment in this clinical investigation, candidates must meet ALL of
the following criteria:
Patients:
• recently diagnosed with PD (N=625; time since Parkinson diagnosis <= 2 years)
or not recently diagnosed with PD (N=625; time since Parkinson diagnosis >2 &
<=15 years) (Time since Parkinson diagnosis (in years) made by a neurologist; In
order to obtain a good representation of the PD population an even distribution
with respect to gender and age (age categories: <55 years, 55-65 years and >65
years) in both patient groups, will be attempted.
• 18 years or older;
• able to read and speak Dutch
• Providing IRB-approved Informed Consent;
• Willing, competent and able to comply with all aspects of the protocol,
including follow-up schedule and biospecimen collections.
Controls:
• 18 years or older
• Healthy (Self-report)
• Is able to read and speak Dutch
• Even distribution with respect to gender and age of the patient groups will
be attempted
• Providing IRB-approved Informed Consent;
• Willing, competent and able to comply with all aspects of the protocol,
including follow-up schedule and biospecimen collections.
Exclusion criteria
Patients
• Patients who received brain surgery for Parkinson*s disease, patients who
currently use levodopa continuous intestinal gel or patients who are currently
receiving apomorphine treatment.
• presence of co-morbidities that would hamper interpretation of parkinsonian
disability, in the opinion of the lnvestigator.
• MoCa score of <=16 (indicates dementia)
• Unwillingness to be informed of unexpected medical findings
Controls
• A history of neurological disorders that affect the brain or central nervous
system
• Abnormal findings at general neurological examination
• Unwillingness to be informed of unexpected medical findings
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL70698.029.19 |
| Other | NL9788 |