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A Post Marketing Observational Study of Activities of Daily Living in Advanced Parkinson's Disease Patients With Early Troublesome Motor Fluctuations and Treated With Duodopa - a Multi-country Study (MONOTREAT)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
AbbVie ( AbbVie (prior sponsor, Abbott) )
ClinicalTrials.gov Identifier:
NCT01747655
First received: December 10, 2012
Last updated: October 6, 2016
Last verified: October 2016
  Purpose
Patients with advanced Parkinson's Disease experience a range in the severity of their motor fluctuations. The rationale for this Post Marketing Observational Study is to demonstrate the benefits of Duodopa treatment on Parkinson's Disease patients entering the advanced stage of the disease whose motor fluctuations have become troublesome and complicate management with oral therapy. The aim of this post-marketing observational study is to assess the effect of Duodopa treatment on activities of daily living in advanced Parkinson's Disease participants characterised by either 2-4 hours of "off" time or 2 hours of non-troublesome or troublesome dyskinesia daily, supported by a Unified Parkinson's Disease Rating Scale Total Score in the best "on" state of at least 40 points at baseline.

Condition
Parkinson's Disease

Study Type: Observational
Study Design: Time Perspective: Prospective
Official Title: A Post Marketing Observational Study of Activities of Daily Living in Advanced Parkinson's Disease Patients With Early Troublesome Motor Fluctuations and Treated With Duodopa - a Multi-country Study - MONOTREAT

Resource links provided by NLM:


Further study details as provided by AbbVie:

Primary Outcome Measures:
  • Unified Parkinson's Disease Rating Scale (UPDRS) II (Activities of Daily Living) Score: Mean Change From Baseline to 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    The UPDRS is an Investigator-used rating tool to follow the longitudinal course of Parkinson's disease. The Part II score is the sum of the answers to 13 questions, each of which are measured on a 5-point scale (0-4). The Part II score ranges from 0-52 and higher scores are associated with more disability. UPDRS scores during "On" time (when PD symptoms are well controlled by the drug) are presented. Last observation carried forward (LOCF) was used for missing data.


Secondary Outcome Measures:
  • Unified Parkinson's Disease Rating Scale (UPDRS) II (Activities of Daily Living) Score: Mean Change From Baseline to 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    The UPDRS is an Investigator-used rating tool to follow the longitudinal course of Parkinson's disease. The Part II score is the sum of the answers to 13 questions, each of which are measured on a 5-point scale (0-4). The Part II score ranges from 0-52 and higher scores are associated with more disability. UPDRS scores during "On" time (when PD symptoms are well controlled by the drug) are presented. n=the number of participants with data at baseline and given time point.

  • Percentage of Participants Who Continued With Jejunal Extension Tube of the Percutaneous Endoscopic Gastrostomy (PEG-J) Treatment [ Time Frame: 14 days ] [ Designated as safety issue: No ]
    The percentage of participants who continued with PEG-J treatment after treatment via temporary naso-jejunal tube.

  • Primary Reasons for Discontinuing Duodopa Treatment or for Discontinuing the Study [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    The primary reasons for stopping treatment with Duodopa or for discontinuing the study.

  • Unified Parkinson's Disease Rating Scale (UPDRS) IV (Complications of Therapy) Item 32 (Duration) Score: Mean Change From Baseline to 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    The UPDRS IV questionnaire consists of 4 individual items that assess the degree of dyskinesias (Item 32: duration; Item 33: disability; and Item 34: pain) and clinical fluctuations (Item 39: percentage of "off" times of the waking day). Individual UPDRS IV item scores range from 0 to 4. Higher scores indicate a higher complication of therapy. Observed values are presented for each visit as well as LOCF at 12 months after discharge. n=the number of participants with data at baseline and given time point.

  • Unified Parkinson's Disease Rating Scale (UPDRS) IV (Complications of Therapy) Item 33 (Disability) Score: Mean Change From Baseline to 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    The UPDRS IV questionnaire consists of 4 individual items that assess the degree of dyskinesias (Item 32: duration; Item 33: disability; and Item 34: pain) and clinical fluctuations (Item 39: percentage of "off" times of the waking day). Individual UPDRS IV item scores range from 0 to 4. Higher scores indicate a higher complication of therapy. Observed values are presented for each visit as well as LOCF at 12 months after discharge. n=the number of participants with data at baseline and given time point.

  • Unified Parkinson's Disease Rating Scale (UPDRS) IV (Complications of Therapy) Item 34 (Pain) Score: Mean Change From Baseline to 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    The UPDRS IV questionnaire consists of 4 individual items that assess the degree of dyskinesias (Item 32: duration; Item 33: disability; and Item 34: pain) and clinical fluctuations (Item 39: percentage of "off" times of the waking day). Individual UPDRS IV item scores range from 0 to 4. Higher scores indicate a higher complication of therapy. Observed values are presented for each visit as well as LOCF at 12 months after discharge. n=the number of participants with data at baseline and given time point.

  • Unified Parkinson's Disease Rating Scale (UPDRS) IV (Complications of Therapy) Item 39 (Clinical Fluctuations) Score: Mean Change From Baseline to 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    The UPDRS IV questionnaire consists of 4 individual items that assess the degree of dyskinesias (Item 32: duration; Item 33: disability; and Item 34: pain) and clinical fluctuations (Item 39: percentage of "off" times of the waking day). Individual UPDRS IV item scores range from 0 to 4. Higher scores indicate a higher complication of therapy. Observed values are presented for each visit as well as LOCF at 12 months after discharge. n=the number of participants with data at baseline and given time point.

  • Unified Parkinson's Disease Rating Scale (UPDRS) III (Motor Examination) Score: Mean Change From Baseline to 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    The UPDRS III questionnaire consists of 14 questions on motor examinations rated from 0 (absent/normal) to 4 (extreme impairment). Questions 20-26 are multi-part questions in that they are evaluated separately for multiple body parts (for example, for the left and right hand). Counting each of these assessments leads to a total of 27 answers. The UPDRS III score ranges from 0 to 108 with higher values indicating greater impairment and was calculated as the sum of the 27 answers provided to the 14 questions. Observed values are presented for each visit as well as LOCF at 12 months after discharge. n=the number of participants with data at baseline and given time point.

  • Non-Motor Symptoms Assessment Scale for Parkinson's Disease (NMSS Rating Scale) Total Score: Mean Change From Baseline to 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    Non-motor symptoms assessed over the previous month were scored with respect to severity (0 = none, 1 = mild, 2 = moderate, 3 = severe) and with respect to frequency (1 = rarely, 2 = often, 3 = frequent, 4 = very frequent). The total NMSS score ranges from 0 to 360 with higher values indicating greater impairment and was calculated as the sum of all individual score values. Observed values are presented for each visit as well as LOCF at 12 months after discharge. n=the number of participants with data at baseline and given time point.

  • Parkinson's Disease Quality of Life Questionnaire (PDQ-8) Summary Index Score: Mean Change From Baseline to 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0), at discharge from hospital, and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    Participants were asked to state how often they had encountered certain problems over the past four weeks using the following rating scale: Never (0), occasionally (1), sometimes (2), often (3), always or cannot do at all (4). The PDQ-8 summary index was derived as the sum of the single items divided by 32. Scores range from 0 to 100. A higher summary index score indicates a higher impairment of quality of life. Observed values are presented for each visit as well as LOCF at 12 months after discharge. n=the number of participants with data at baseline and given time point.

  • Healthcare Resource Utilization (HCRU) Number of Office Visits: Mean Change From Baseline to 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    Participants were asked about their utilization of healthcare resources within the previous 3 months, including total number of office visits, total number of visits at home for Parkinson's disease, total number of emergency situations (overnight hospital stay, visit at emergency room, calls for immediate assistance [family/friend)], and calls to 911/emergency), received assistance at home (family/friend or paid caregiver), and falls. n=the number of participants with data at baseline and given time point.

  • Healthcare Resource Utilization (HCRU) Number of Visits at Home: Mean Change From Baseline to 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    Participants were asked about their utilization of healthcare resources within the previous 3 months, including total number of office visits, total number of visits at home for Parkinson's disease, total number of emergency situations (overnight hospital stay, visit at emergency room, calls for immediate assistance [family/friend)], and calls to 911/emergency), received assistance at home (family/friend or paid caregiver), and falls. n=the number of participants with data at baseline and given time point.

  • Healthcare Resource Utilization (HCRU) Emergency Situations: Percentage of Participants With Emergency Situations at 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    Participants were asked about their utilization of healthcare resources within the previous 3 months, including total number of office visits, total number of visits at home for Parkinson's disease, total number of emergency situations (overnight hospital stay, visit at emergency room, calls for immediate assistance [family/friend)], and calls to 911/emergency), received assistance at home (family/friend or paid caregiver), and falls. n=the number of participants with data at given time point.

  • Healthcare Resource Utilization (HCRU) Received Assistance at Home: Percentage of Participants Who Received Assistance at Home at 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    Participants were asked about their utilization of healthcare resources within the previous 3 months, including total number of office visits, total number of visits at home for Parkinson's disease, total number of emergency situations (overnight hospital stay, visit at emergency room, calls for immediate assistance [family/friend)], and calls to 911/emergency), received assistance at home (family/friend or paid caregiver), and falls. n=the number of participants with data at given time point.

  • Healthcare Resource Utilization (HCRU) Falls: Percentage of Participants With Falls at 3, 6, and 12 Months After Hospital Discharge [ Time Frame: Baseline (Week 0) and 3, 6, and 12 months after hospital discharge ] [ Designated as safety issue: No ]
    Participants were asked about their utilization of healthcare resources within the previous 3 months , including total number of office visits, total number of visits at home for Parkinson's disease, total number of emergency situations (overnight hospital stay, visit at emergency room, calls for immediate assistance [family/friend)], and calls to 911/emergency), received assistance at home (family/friend or paid caregiver), and falls. n=the number of participants with data at baseline and given time point.


Enrollment: 64
Study Start Date: February 2013
Study Completion Date: July 2015
Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
Groups/Cohorts
Duodopa
Participants given Duodopa gel administered with a portable pump directly into the proximal small intestine by a jejunal extension tube of the percutaneous endoscopic gastrostomy (PEG-J)
Standard of Care
Participants that return to oral or transdermal anti-parkinson's disease medications

Detailed Description:

Data are recorded from visits most closely aligned with the planned periods of Visit 0 (V0): Baseline (After decision to use the temporary naso-duodenal tube (approximately 2-14 days) and after signature of the Patient Authorization/Informed Consent Form); Visit 1 (V1): At discharge from hospital; Visit 2 (V2): 3 months after discharge; Visit 3 (V3): 6 months after discharge; Visit 4 (V4): 12 months after discharge.

All participants have a temporary naso-duodenal tube used initially with the infusion pump to determine if the participant responds favorably to this method of treatment and to optimize the dose of Duodopa before permanent treatment is started.

Participants who choose a treatment other than Duodopa after the temporary naso-duodenal test phase are considered for the Standard of Care group. Participants who go on to select Apomorphine pump or Deep Brain Stimulation at any stage are not eligible to continue in this group or to continue in the observational period of study.

Participants who continue to with Duodopa treatment after the the temporary naso-duodenal test phase are the Duodopa group.

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Hospital clinic
Criteria

Inclusion Criteria:

  • Has advanced levodopa-responsive Parkinson's disease
  • The decision to treat with Duodopa is made by the physician in accordance with the local label (Summary of Product Characteristics; product label) prior to any decision to approach the patient to participate in this study
  • Parkinson's Disease (PD) medicinal treatment is unchanged for at least four weeks prior to baseline
  • Takes oral medication four or more times daily
  • Either has 2-4 hours of "off" time or 2 hours of non-troublesome or troublesome dyskinesia daily supported by a Unified Parkinson's Disease Rating Scale (UPDRS) Total Score in the best "on" state of at least 40 points at baseline; based on documented medical history

Exclusion Criteria:

  • Use of Deep Brain Stimulation (DBS), Apomorphine pump or Duodopa treatment prior to baseline visit
  • Severe dementia based on a Mini-Mental State Examination (MMSE) of < 24
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01747655

Sponsors and Collaborators
AbbVie (prior sponsor, Abbott)
Investigators
Study Director: Koray Onuk, MD AbbVie
  More Information

Additional Information:
Responsible Party: AbbVie (prior sponsor, Abbott)
ClinicalTrials.gov Identifier: NCT01747655     History of Changes
Other Study ID Numbers: P13-893 
Study First Received: December 10, 2012
Results First Received: July 21, 2016
Last Updated: October 6, 2016
Health Authority: Germany: Federal Institute for Drugs and Medical Devices
Italy: The Italian Medicines Agency
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Austria: Agency for Health and Food Safety

Keywords provided by AbbVie:
Early Observational Advanced
Parkinson's Disease

Additional relevant MeSH terms:
Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases
Carbidopa, levodopa drug combination
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs
Antiparkinson Agents
Anti-Dyskinesia Agents
Dopamine Agonists
Dopamine Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on December 05, 2016