A Study to Investigate a New Treatment in Patients With Parkinson's Disease
- Conditions
- Parkinson Disease
- Interventions
- Genetic: Optional pharmacogenetic assessmentOther: QuestionnairesOther: Optional Blood-Oxygen-level Dependent functionalMRIOther: Motor Assessments on regular PD treatmentOther: Motor Assessments before taking regular PD treatment
- Registration Number
- NCT03407378
- Lead Sponsor
- Tools4Patient
- Brief Summary
The purpose of this clinical trial conducted in patients with Parkinson's Disease is to study the relationship between patient individual profile and their response to IPT803 Adjunct Treatment (treatment response being characterized by movements improvement).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 110
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Assessments ON regular PD treatment Optional Blood-Oxygen-level Dependent functionalMRI IPT803 Questionnaires Motor assessments on regular PD treatment Optional pharmacogenetic assessments Optional Blood-Oxygen-level Dependent Functional-MRI Assessments ON regular PD treatment Optional pharmacogenetic assessment IPT803 Questionnaires Motor assessments on regular PD treatment Optional pharmacogenetic assessments Optional Blood-Oxygen-level Dependent Functional-MRI Assessments ON regular PD treatment Questionnaires IPT803 Questionnaires Motor assessments on regular PD treatment Optional pharmacogenetic assessments Optional Blood-Oxygen-level Dependent Functional-MRI Assessments OFF regular PD treatment Optional Blood-Oxygen-level Dependent functionalMRI IPT803 Questionnaires Motor assessments before taking regular PD treatment Optional pharmacogenetic assessments Optional Blood-Oxygen-level Dependent Functional-MRI Assessments ON regular PD treatment Motor Assessments on regular PD treatment IPT803 Questionnaires Motor assessments on regular PD treatment Optional pharmacogenetic assessments Optional Blood-Oxygen-level Dependent Functional-MRI Assessments OFF regular PD treatment Questionnaires IPT803 Questionnaires Motor assessments before taking regular PD treatment Optional pharmacogenetic assessments Optional Blood-Oxygen-level Dependent Functional-MRI Assessments OFF regular PD treatment Motor Assessments before taking regular PD treatment IPT803 Questionnaires Motor assessments before taking regular PD treatment Optional pharmacogenetic assessments Optional Blood-Oxygen-level Dependent Functional-MRI Assessments OFF regular PD treatment Optional pharmacogenetic assessment IPT803 Questionnaires Motor assessments before taking regular PD treatment Optional pharmacogenetic assessments Optional Blood-Oxygen-level Dependent Functional-MRI Assessments ON regular PD treatment IPT803 IPT803 Questionnaires Motor assessments on regular PD treatment Optional pharmacogenetic assessments Optional Blood-Oxygen-level Dependent Functional-MRI Assessments OFF regular PD treatment IPT803 IPT803 Questionnaires Motor assessments before taking regular PD treatment Optional pharmacogenetic assessments Optional Blood-Oxygen-level Dependent Functional-MRI
- Primary Outcome Measures
Name Time Method Patient's change from baseline of score as measured by Movement Disorder Society-Sponsored Unified Parkinson's Disease Rating Scale Part III (MDS-UPDRS; Part III), after 12 weeks of IPT803 administration. Time zero equals baseline equals (Visit 2 - Day 1 prior to IPT803 first dose) up to Visit 4 (Day 85) Part III of the MDS-UPDRS (or motor examination) assesses the motor abilities in PD patients at the time of the visit. This part measures 18 motor examinations such as speech, facial expression, tremor, rigidity, finger tapping, pronation-supination movements of hands, leg agility, arising from chair, gait.
The qualified rater must score 34 items from 0 to 4, where 0 indicates a normal situation and 4 indicates that PD interferes severely in carrying out the task. The total score, being the sum of all these items, can be between 0 to 136.
- Secondary Outcome Measures
Name Time Method Patient's change from baseline of safety incidence as measured by the rate and severity of Treatment emergent adverse event (TEAEs). Time zero equals baseline Visit 2 IPT803 first dose (Day 1) up to Visit 4 (Day 85) Patient's change from baseline of motor and non-motor outcomes as measured by Part I, Part II and IV subscales of Movement Disorder Society-Sponsored Unified Parkinson's Disease Rating Scale (MDS-UPDRS). From Visit 1 (Day -14 to Day -7) up to Visit 4 (Day 85) The MDS-UPDRS is divided into 4 parts. In each part, all items are rated on a scale from 0 (normal) to 4 (severe impairment).
Part I assesses 15 items of non-motor aspects of experiences of daily living. Part IA is assessed by a qualified rater, Part IB is completed by the patient. The total score, being the sum of all these items, can be between 0 to 60.
Part II comprises 13 items evaluating the impact of PD on patients' activities of daily living (ADL) over the week prior to the visit such as speech, salivation, swallowing, eating, handwriting, dressing, turning in bed, walking. It will be completed by the patient. The total score, being the sum of all these items, can be between 0 to 52.
Part IV assesses motor complications of therapy, such as dyskinesias, motor fluctuations. This part (6 items) is completed by a qualified rater. The total score, being the sum of all these items, can be between 0 to 24.The patient's change from baseline in disease severity as measured by the Parkinson's Disease Questionnaire (PDQ-39). From Visit 2 (Day 1) up to Visit 4 (Day 85) Patient's change from baseline of fatigue as measured by the Fatigue Severity Scale (FSS). From Visit 1 (Day -14 to Day -7) up to Visit 4 (Day 85) The FSS is a self-administered questionnaire with 9 items assessing the severity of fatigue during the past 2 weeks. Grading of each item ranges from 1 to 7, where 1 indicates strong disagreement and 7 strong agreement, where the addition of all numbers circled by patient get the final score.
Patient's change from baseline of sleep quality as measured by the Epworth Sleep Scale (ESS). From Visit 1 (Day -14 to Day -7) up to Visit 4 (Day 85) The ESS assesses the overall level of daytime sleepiness. Eight items describe normative daily situations known to vary in their soporific qualities. Patients rate the likelihood of dozing off or falling asleep. The test is rated on a 4-point scale (0=would never doze off to 3=high chance of dozing off).
Investigator change from baseline in disease severity as measured by the Investigator Assessment of Changes (IGAC). From Visit 2 (Day 1) up to Visit 4 (Day 85) IGAC is a subjective evaluation by the Investigator using a 0 to 10 Numeric Rating Scale (NRS) to answer the following question: "If you take into consideration all the various ways that motor control influences the patient and his/her life, how do you then evaluate the patient's motor condition today?", with 0 meaning "very bad" and 10 "very good".
Patient's change from baseline in disease severity as measured by the Patient Assessment of Changes (PGAC). From Visit 2 (Day 1) up to Visit 4 (Day 85) PGAC is a subjective evaluation by the patient using a 0 to 10 Numeric Rating Scale (NRS) to answer the following question: "If you take into consideration all the various ways that motor control influences you and your life, how do you then evaluate your motor condition over the last week?", with 0 meaning "very bad" and 10 meaning "very good".
Patient's change from baseline above or equal to the minimal clinically important difference (MCID) of the motor score as measured by Part II and III subscales of Movement Disorder Society-Sponsored Unified Parkinson's Disease Rating Scale (MDS-UPDRS). Time zero equals baseline (Visit 2 - Day 1 prior to IPT803 first dose) up to Visit 4 (Day 85) The Minimal Clinically Important Difference (MCID) is the smallest change in an outcome measure that is meaningful for patients.
In the literature, the MCIDs of MDS-UPDRS Part II and Part III are respectively defined as a motor score reduction of 2 and 6 points. The motor scores are the sum of all items (ranging from 0-4) of each MDS-UPDRS subscale.
Following endpoints will be measured:
* A reduction above or equal to 2 points of MDS-UPDRS Part II motor score (minimum score= 0, maximal score= 52)
* A reduction above or equal to 6 points of MDS-UPDRS Part III motor score (minimum score= 0, maximal score= 136)
These endpoints will be interpreted as binary values: 0 (below) and 1 (above or equal).Patient's change from baseline above or equal to 30 % or above or equal to 50 % of the motor score as measured by Part II and III subscales of MDS-UPDRS. Time zero equals baseline (Visit 2 - Day 1 prior to IPT803 first dose) up to Visit 4 (Day 85) The motor scores are the sum of all items (ranging from 0-4) of each MDS-UPDRS subscale.
Following endpoints will be measured:
* A reduction above or equal to 30% of MDS-UPDRS Part II motor score
* A reduction above or equal to 30% of MDS-UPDRS Part III motor score
* A reduction above or equal to 50% of MDS-UPDRS Part II motor score
* A reduction above or equal to 50% of MDS-UPDRS Part III motor score
These endpoints will be interpreted as binary values: 0 (below) and 1 (above or equal).Cronbach α assessment of MPsQ at Visit 1 (Day -14 to Day -7), Visit 2 (Day 1) Visit 4 (Day 85)
Trial Locations
- Locations (10)
University of Colorado School of Medicine
🇺🇸Aurora, Colorado, United States
Northwestern
🇺🇸Chicago, Illinois, United States
Henry Ford
🇺🇸West Bloomfield, Michigan, United States
Columbia
🇺🇸New York, New York, United States
CHU Liege - Liège University
🇧🇪Liege, Belgium
CHU Rennes - Hopital Pontchaillou
🇫🇷Rennes, France
CHU Grenoble
🇫🇷Grenoble, France
CHU Poitiers
🇫🇷Poitiers, France
CHU Purpan - Hopital Pierre Paul Riquet
🇫🇷Toulouse, France
University of Florida
🇺🇸Gainesville, Florida, United States