Clinical trial NCT03407378: A Study to Investigate a New Treatment in Patients With Parkinson's Disease
Official title: A Proof of Concept Study to Investigate the Effect of IPT803 Adjunct Treatment in Patients With Parkinson's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Parkinson Disease
Lead sponsor: Tools4Patient
Sponsor class: OTHER
First posted: 2018-01-23
Enrollment: 110
Sex: ALL
Minimum age (years): 35
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: BASIC_SCIENCE

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).


Eligibility criteria: Inclusion Criteria

1. Men or women of at least 35 years of age;
2. Are reliable and willing to make themselves available for the duration of the study and are willing to follow study procedures;
3. Have given written informed consent approved by the relevant Ethics Committee (EC)/Institutional Review Board (IRB) governing the study site(s);
4. Medically stable outpatients with idiopathic PD based on the MDS-PD criteria (Postuma et al 2015). The diagnosis must be confirmed by bradykinesia plus one of the other cardinal signs (resting tremor, rigidity or postural instability not caused by primary visual, vestibular, cerebellar, or proprioceptive dysfunction) being present, without any other known or suspected cause of Parkinson;
5. Patients with a Hoehn and Yahr Stage \< 3;
6. Patients with a MMSE ≥ 26;
7. Patient stabilized with PD medication(s) e.g. levodopa, dopamine agonists, amantadine and/or Monoamine oxidase (MAO)-B inhibitors for at least 4 weeks prior to Visit 1 and and up to Visit 4 included or Drug naïve patients recently diagnosed with PD according to the criteria above and for whom PD medication(s) may be initiated after Visit 4;

   Exclusion Criteria
8. Pregnant (urine pregnancy test), breastfeeding, or willing to be pregnant during the study;
9. Presence of clinically significant medical or psychiatric condition that may increase the risk associated with study participation or investigational product/device administration or participation in any other type of medical research that may interfere with the interpretation of study results in the judgment of the sponsor/investigator or in an exclusion period according to national law, would make the subject inappropriate for entry into this study;
10. Has a history of psychotic symptoms requiring treatment with a neuroleptic medication within the past 12 months;
11. Any current primary psychiatric condition, including not stabilized mood disorders, personality disorders or mental retardation based on diagnostic following DSM-V;
12. Any known hypersensitivity to corn and/or corn-derived products;
13. Alcohol dependence or regular use of known drugs of abuse (e.g., amphetamines, barbiturates, benzodiazepines, cannabinoids, cocaine, methadone, opiates, and phencyclidine);
14. Any other relevant medical disorder/acute disease state judged by the Investigator as likely to interfere with study procedures or represent a risk for the patient;
15. Any close relationship with the investigators or employees or consultants of the sponsor (i.e. belonging to immediate family or subordination relationship);
16. Under legal protection, according to the national law (for French sites only);
17. Are persons who have previously received IPT803, have completed or withdrawn from this study or any other study investigating IPT803.
18. Change in the patient's regular PD medication(s) (dosage or dosing interval) or introduction of a new regular PD medication(s) within 4 weeks prior to Visit 1 and up to Visit 4 included;
19. Patients with motor complications (wearing off; dyskinesia) that would interfere with study procedures;
20. Patients with history or clinical features consistent with an atypical Parkinsonian syndrome (for example: supranuclear gaze palsy, clinically significant orthostatic hypotension);
21. History of surgical or invasive intervention for PD (pallidotomy, thalamotomy, deep brain stimulation, etc.);
22. Any Parkinson's disease-related feature or symptom that could interfere with the study conduct and results as assessed by the investigator.

    Exclusion Imaging (BOLD fMRI) criteria for patients following Imaging Procedure:
23. Patients unable to undergo MRI scans, including suffering from claustrophobia;
24. Have implanted or embedded metal objects or fragments in the head or body that would present a risk during the MRI scanning procedure, or have worked with ferrous metals either as a vocation or hobby (for example, as a sheet metal worker, welder, or machinist) in such a way that might have led to unknown, indwelling metal fragments that could cause injury if they moved in response to placement in the magnetic field according to investigator site judgment.

Primary outcomes:
measure: 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.
timeFrame: Time zero equals baseline equals (Visit 2 - Day 1 prior to IPT803 first dose) up to Visit 4 (Day 85)
Secondary outcomes:
measure: Patient's change from baseline of safety incidence as measured by the rate and severity of Treatment emergent adverse event (TEAEs).
timeFrame: Time zero equals baseline Visit 2 IPT803 first dose (Day 1) up to Visit 4 (Day 85)
measure: 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).
timeFrame: From Visit 1 (Day -14 to Day -7) up to Visit 4 (Day 85)
measure: The patient's change from baseline in disease severity as measured by the Parkinson's Disease Questionnaire (PDQ-39).
timeFrame: From Visit 2 (Day 1) up to Visit 4 (Day 85)
measure: Patient's change from baseline of fatigue as measured by the Fatigue Severity Scale (FSS).
timeFrame: From Visit 1 (Day -14 to Day -7) up to Visit 4 (Day 85)
measure: Patient's change from baseline of sleep quality as measured by the Epworth Sleep Scale (ESS).
timeFrame: From Visit 1 (Day -14 to Day -7) up to Visit 4 (Day 85)
measure: Investigator change from baseline in disease severity as measured by the Investigator Assessment of Changes (IGAC).
timeFrame: From Visit 2 (Day 1) up to Visit 4 (Day 85)
measure: Patient's change from baseline in disease severity as measured by the Patient Assessment of Changes (PGAC).
timeFrame: From Visit 2 (Day 1) up to Visit 4 (Day 85)
measure: 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).
timeFrame: Time zero equals baseline (Visit 2 - Day 1 prior to IPT803 first dose) up to Visit 4 (Day 85)
measure: 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.
timeFrame: Time zero equals baseline (Visit 2 - Day 1 prior to IPT803 first dose) up to Visit 4 (Day 85)
measure: Cronbach α assessment of MPsQ
timeFrame: at Visit 1 (Day -14 to Day -7), Visit 2 (Day 1) Visit 4 (Day 85)
Other outcomes:
measure: Patient's change from baseline of motor score, as measured by Inertial Measurement Unit (IMU) on Finger taping (FT) and Pronation-supination movement of the hands (PSH) during the 12-week treatment.
timeFrame: Time zero equals baseline equals (Visit 2 - Day 1 prior to IPT803 first dose) up to Visit 4 (Day 85)
measure: Patient's change from baseline of the regional brain activity as measured by BOLD fMRI measured by motor tasks performed during fMRI before and after single dose of IPT803.
timeFrame: On Day 1: prior to IPT 803 first dose and 60 minutes after IPT803 first dose
measure: Patient's change of motor score, as measured by Part III subscale of MDS-UPDRS directly after IPT803 single dose.
timeFrame: On Day 1: prior to IPT 803 first dose and 30 minutes after IPT803 first dose
Locations:
city: Aurora
country: United States
facility: University of Colorado School of Medicine
state: Colorado
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Chicago
country: United States
facility: Northwestern
state: Illinois
city: West Bloomfield
country: United States
facility: Henry Ford
state: Michigan
city: New York
country: United States
facility: Columbia
state: New York
city: Liege
country: Belgium
facility: CHU Liege - Liège University
city: Grenoble
country: France
facility: CHU Grenoble
city: Poitiers
country: France
facility: CHU Poitiers
city: Rennes
country: France
facility: CHU Rennes - Hopital Pontchaillou
city: Toulouse
country: France
facility: CHU Purpan - Hopital Pierre Paul Riquet
Study officials:
affiliation: Tools4Patient
name: Pereira Alvaro
role: STUDY_DIRECTOR
References: