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出境医 / 临床实验 / Evaluation of Clinical Progression in Patients With Motor Predominant Parkinson Disease. (PARK001)

Evaluation of Clinical Progression in Patients With Motor Predominant Parkinson Disease. (PARK001)

Study Description
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.

Condition or disease
Parkinson Disease

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.

Study Design
Layout table for study information
Study Type : Observational
Estimated Enrollment : 30 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Longitudinal Exploratory Prospective Evaluation of Clinical Evolution in Patients With Motor Predominant Parkinson Disease.
Actual Study Start Date : January 14, 2019
Estimated Primary Completion Date : October 30, 2022
Estimated Study Completion Date : October 30, 2022
Arms and Interventions
Outcome Measures
Primary Outcome Measures :
  1. Change from baseline in Movement Disorders Society Unified Parkinson's Disease Rating Scale Part 3 (MDS-UPDRS Part III) "off" medications [ Time Frame: baseline, 6, 12, 18, 24 and 30 months after baseline ] ]
    An examiner will measure MDS-UPDRS part 3 motor scores "off" medications. MDS-UPDRS Part III is a motor examination consisting of 18 summed items where the investigator rates each motor symptom based on a scale of 0 - 4, higher values indicating worse function.


Secondary Outcome Measures :
  1. Change from Baseline of brain MRI [ Time Frame: 12 and 24 months ]
    neuromelanin sequence


Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   up to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patient with motor predominant PD aged 70 or less, with a disease onset between 2-8 years.
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)
Contacts and Locations

Locations
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France
Hôpital Henri Mondor
Créteil, France
Hôpital Pitié-Salpétrière
Paris, France
Sponsors and Collaborators
BrainEver
Tracking Information
First Submitted Date May 10, 2019
First Posted Date May 23, 2019
Last Update Posted Date September 21, 2020
Actual Study Start Date January 14, 2019
Estimated Primary Completion Date October 30, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 21, 2020) 		Change from baseline in Movement Disorders Society Unified Parkinson's Disease Rating Scale Part 3 (MDS-UPDRS Part III) "off" medications [ Time Frame: baseline, 6, 12, 18, 24 and 30 months after baseline ] ]
An examiner will measure MDS-UPDRS part 3 motor scores "off" medications. MDS-UPDRS Part III is a motor examination consisting of 18 summed items where the investigator rates each motor symptom based on a scale of 0 - 4, higher values indicating worse function.
Original Primary Outcome Measures
 (submitted: May 22, 2019) 		Change from baseline in Movement Disorders Society Unified Parkinson's Disease Rating Scale Part 3 (MDS-UPDRS Part III) "off" medications [ Time Frame: baseline, 6, 12 and 18 months after baseline ] ]
An examiner will measure MDS-UPDRS part 3 motor scores "off" medications. MDS-UPDRS Part III is a motor examination consisting of 18 summed items where the investigator rates each motor symptom based on a scale of 0 - 4, higher values indicating worse function.
Change History
Current Secondary Outcome Measures
 (submitted: April 21, 2020) 		Change from Baseline of brain MRI [ Time Frame: 12 and 24 months ]
neuromelanin sequence
Original Secondary Outcome Measures
 (submitted: May 22, 2019) 		Change from Baseline of brain MRI [ Time Frame: 12-months ]
neuromelanin sequence
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title 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.
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.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Patient with motor predominant PD aged 70 or less, with a disease onset between 2-8 years.
Condition Parkinson Disease
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Suspended
Estimated Enrollment
 (submitted: May 22, 2019) 		30
Original Estimated Enrollment Same as current
Estimated Study Completion Date October 30, 2022
Estimated Primary Completion Date October 30, 2022   (Final data collection date for primary outcome measure)
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)
Sex/Gender
Sexes Eligible for Study: All
Ages up to 70 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries France
Removed Location Countries  
 
Administrative Information
NCT Number NCT03960242
Other Study ID Numbers BREN01_CLIN_001
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party BrainEver
Study Sponsor BrainEver
Collaborators Not Provided
Investigators Not Provided
PRS Account BrainEver
Verification Date September 2020

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