Clinical trial NCT03960242: Evaluation of Clinical Progression in Patients With Motor Predominant Parkinson Disease.
Official title: Longitudinal Exploratory Prospective Evaluation of Clinical Evolution in Patients With Motor Predominant Parkinson Disease.
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Parkinson Disease
Lead sponsor: BrainEver
Sponsor class: INDUSTRY
First posted: 2019-05-23
Enrollment: 3
Sex: ALL
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patient with motor predominant PD aged 70 or less, with a disease onset between 2-8 years.

Brief summary: The objective of this study is the longitudinal prospective clinical evaluation in patients with motor predominant PD; it will assess the individual disease progression (change) of the clinical and imaging parameters measured at 6-month interval over a minimum of 12 months.

Detailed description: The absence of clear PD subtypes defining a common path and rate of disease progression, combined with the absence of validated biomarkers indicative of disease progression and a strong placebo effect, constitute a challenge for clinical trials assessing new potential disease-modifying therapies in PD.

The present cohort would constitute a well-defined population of patients, with individual progression assessment, suitable to evaluate new disease-modifying therapies in a subsequent Phase1/2 clinical trial.

Eligibility criteria: Inclusion Criteria:

1. Idiopathic Parkinson's Disease (According to British Brain Bank Criteria); genetic forms of PD are not excluded
2. Male or female adult, aged 70 or less
3. Modified Hoehn and Yahr stage 2 to 3 in OFF state
4. Disease duration, i.e. time from first motor symptoms between 2 to 8 (included) years ; first motor symptoms to be taken into consideration being the cardinal motor symptoms of PD (bradykinesia, tremor and rigidity)
5. Univoqual response to L-DOPA (of at least 50% in MDS-UPDRS Part III motor score)
6. Presence of fluctuations and/or dyskinesia
7. MDS-UPDRS total motor score ≥15 in OFF state
8. L-Dopa treatment stable for at least 4 weeks
9. Covered by healthcare insurance
10. Written informed consent form signed

Exclusion Criteria:

1. Scan Without Evidence of Dopamine Deficit (SWEDD) (DaTSCAN)
2. Atypical parkinsonism syndrome
3. Dementia as detected by a score \< 21/30 at the Montreal Cognitive Assessment Screening (MoCA)
4. Psychiatric disorders including major depression with suicidal thoughts as evaluated by a psychiatrist or a neurologist at the selection period
5. Any medical or psychological problems which may interfere with a smooth conduction of the study protocol (e.g. cancer with a limited life expectancy)
6. History of significant brain or vascular disease (tumor, epilepsy, stroke ...)
7. Any contraindication for undergoing MRI of the head
8. Prior brain surgical procedures with or without implementation of an intra-cerebral device
9. Drug or alcohol addiction
10. Pregnancy or breastfeeding
11. Patient with reproductive potential who do not agree to use an accepted effective method of contraception - investigator's judgment- during the study period
12. Illiteracy or insufficient language skills (French) to complete the questionnaires
13. Patient deprived of liberty by a judicial or administrative decision, or who is under a measure of legal protection (e.g. guardianship or curatorship).
14. Simultaneous participation in another clinical trial with the administration of investigational drug(s)

Primary outcomes:
measure: Change from baseline in Movement Disorders Society Unified Parkinson's Disease Rating Scale Part 3 (MDS-UPDRS Part III) "off" medications
timeFrame: baseline, 6, 12, 18, 24 and 30 months after baseline ]
Secondary outcomes:
measure: Change from Baseline of brain MRI
timeFrame: 12 and 24 months
Other outcomes:
Locations:
city: Créteil
country: France
facility: Hôpital Henri Mondor
city: Paris
country: France
facility: Hôpital Pitié-Salpétrière
Study officials:
References: