Idiopathic Parkinson Disease
Conditions
Keywords
Parkinson's disease, Safinamide, Xadago, MAO-B inhibitor, off, non-motor symptoms, motor symptoms, levodopa, PD
Brief summary
This is a Phase IV, prospective, observational, post-marketing study designed to obtain additional data on the effect of XADAGO on motor and non-motor symptoms in Parkinson's Disease patients newly prescribed XADAGO.
Detailed description
This Phase IV, multicenter, prospective, observational study to evaluate clinician-reported outcomes and patient-reported outcomes related to motor and non-motor symptoms, health status, quality of life and treatment satisfaction in PD patients who have been newly prescribed XADAGO according to Package Insert indication. This study also will gather real world data from a PD population in the US regarding their overall experience and degree of satisfaction with the use of XADAGO as an add-on treatment to their L-dopa regimen. Treatment experience will be captured using patient self-rating assessments as well as clinician ratings on assessments.
Interventions
DRUGXADAGO (safinamide)
XADAGO (safinamide) is a monoamine oxidase type B (MAO-B) inhibitor indicated as adjunctive treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing off episodes.
Sponsors
Supernus Pharmaceuticals, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
30 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Patient (and Care Partner, if required per Inclusion Criterion 6) is able to understand and provide signed informed consent and HIPAA authorization in English. 2. Patient with diagnosis of idiopathic PD (all stages). 3. Independent of the study, clinician's and patient's choice of treatment is XADAGO in accordance with the Package Insert indication. 4. Patient is willing and able to participate in the study and complete study-related assessments for 2 months and, patients can continue for an optional 4-month study extension. 5. Patient has access to an electronic device for the interim completion of PROs. 6. Patient has an available Care Partner who is able and willing to assist with clinic attendance and completion of study assessments (e.g., PROs, health outcomes, etc.), if in the PI's opinion, assistance is needed to comply with all study visits and procedures.
Exclusion criteria
1. Any of the warnings, precautions, or contraindications listed in the XADAGO Package Insert that in the opinion of the PI would prevent appropriate treatment with XADAGO or impair study participation (e.g., pregnancy, lactation, severe hepatic impairment, etc.). 2. Participation in any other clinical trial of an investigational drug or device within 4 weeks prior to the Baseline Visit or at any time during the study. 3. Patient is currently receiving chemotherapy or radiation for any form of cancer (if history of cancer, must be in clinical remission at study entry) or currently receiving immunotherapy. 4. Patients with conditions that are likely to prevent them from accurately and reliably completing study assessments, including evidence of moderate or severe dementia as determined by the clinician (not to include mild cognitive impairment \[MCI\]); major psychiatric illness (specifically diagnosis of schizophrenia, bipolar disorder or a history of attempted suicide); and/or severe and progressive medical illness (including terminal cancer, end-stage renal disease +/- undergoing dialysis). 5. Severe or unpredictable dyskinesia at the time of the Baseline Visit. 6. Previous participation in this study; a patient may not re-enroll after prior discontinuation or completion
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Baseline to Study Day 60 | MDS-UPDRS: 4-part assessment of the multiple clinical disabilities of Parkinson's Disease. Part I (13 items; Score 0-52) examines non-motor experiences, Part II (13 items; Score 0-52) examines motor experiences, Part III (33 items; Score 0-132) examines the cardinal motor disabilities and Part IV (6 items; Score 0-24) examines motor complications. Each Part has 0-4 ratings, where 0 (no problems) to 4 (severe problems) and scores for each part are summed to calculate the total score which ranges from 0-260. Higher scores represent worse outcomes for each part and total score. |
| Change From Baseline in Parkinson's Disease Questionnaire (PDQ-39) Scores | Baseline to Study Day 60 | PDQ-39 is a patient-reported outcome designed to address aspects of functioning and well-being for those affected by PD. Each of the 39 items is rated using a 5 point Likert scale with 0 for never having difficulties/problems and 4 for always having difficulties/problems. The sum score of the 39 items will be calculated and used for analysis, with scores ranging from 0-156. Higher scores indicate worse outcomes. |
| Change From Baseline in Montreal Cognitive Assessment (MoCA) Total Score. | Baseline to Study Day 60 | MoCA is a 30-point, 1-page test designed to assess several cognitive domains, including visuospatial abilities (5 points), naming (3 points), attention (6 points), language (3 points), abstraction (2 points), delayed recall (5 points), and orientation to time and place (6 points). The total score ranges from 0 to 30, with higher scores indicating better performances. |
| Treatment Satisfaction Questionnaire for Medication (TSQM-9) Scores | Study Day 60 | TSQM-9 consists of 9 questions to assess patients' satisfaction with medication using a range of responses from 1 (extremely dissatisfied) to (7 extremely satisfied). This patient reported outcome provides scores on three parts: effectiveness, convenience, and global satisfaction. The sum of the 9-questions will be calculated and used for analysis. The total score ranges from 0 to 63, with higher scores indicating better treatment satisfaction. |
| Clinical Global Impression of Change (CGI-C) | Study Day 60 | CGI-C is a 7-point scale depicting a Principal Investigator or certified Health Care Professional designee rating of the patient's overall improvement using a range of responses from a minimum of 1 (very much improved) to a maximum of 7 (very much worse). |
| Patient Global Impression of Change (PGI-C) | Study Day 60 | PGI-C is a 7-point scale depicting a patient's rating of overall improvement using a range of responses from 1 (very much improved) to 7 (very much worse). |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| DA Switchers Parkinson's Disease patients that have switched to safinamide (XADAGO) from a dopamine agonist (DA) | 2 |
| MAO-B Parkinson's Disease patients that have switched to safinamide (XADAGO) from a monoamine oxidase-B (MAO-B) inhibitor | 34 |
| MAO-B Naive Parkinson's Disease patients taking safinamide (XADAGO) that are MAO-B inhibitor naïve. | 128 |
| Total | 164 |
Baseline characteristics
| Characteristic | DA Switchers | MAO-B | MAO-B Naive | Total |
|---|---|---|---|---|
| Age, Continuous | 62.5 years STANDARD_DEVIATION 6.36 | 65.3 years STANDARD_DEVIATION 8.28 | 68.1 years STANDARD_DEVIATION 7.77 | 67.4 years STANDARD_DEVIATION 7.92 |
| Age, Customized Age 30-65 | 1 Participants | 16 Participants | 39 Participants | 56 Participants |
| Age, Customized Age >= 65 | 1 Participants | 18 Participants | 89 Participants | 108 Participants |
| BMI (kg/m^2) | 29.9 kg/m^2 STANDARD_DEVIATION 2.36 | 28.4 kg/m^2 STANDARD_DEVIATION 5.17 | 30.1 kg/m^2 STANDARD_DEVIATION 7.05 | 29.7 kg/m^2 STANDARD_DEVIATION 6.68 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 6 Participants | 6 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 2 Participants | 34 Participants | 122 Participants | 158 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Height (cm) | 179.6 cm STANDARD_DEVIATION 6.51 | 173.1 cm STANDARD_DEVIATION 11.67 | 171.0 cm STANDARD_DEVIATION 11.91 | 171.6 cm STANDARD_DEVIATION 11.83 |
| Movement Disorders Society - Unified Parkinson's Disease Rating Scale Part 1 | 12.0 units on a scale STANDARD_DEVIATION 2.83 | 10.5 units on a scale STANDARD_DEVIATION 6.32 | 12.3 units on a scale STANDARD_DEVIATION 6.88 | 11.9 units on a scale STANDARD_DEVIATION 6.75 |
| Movement Disorders Society - Unified Parkinson's Disease Rating Scale Part 2 | 7.0 units on a scale STANDARD_DEVIATION 4.24 | 11.8 units on a scale STANDARD_DEVIATION 9.21 | 13.8 units on a scale STANDARD_DEVIATION 8.66 | 13.3 units on a scale STANDARD_DEVIATION 8.75 |
| Movement Disorders Society - Unified Parkinson's Disease Rating Scale Part 3 | 24.0 units on a scale STANDARD_DEVIATION 2.83 | 23.4 units on a scale STANDARD_DEVIATION 14.13 | 30.0 units on a scale STANDARD_DEVIATION 26 | 28.7 units on a scale STANDARD_DEVIATION 15.95 |
| Movement Disorders Society - Unified Parkinson's Disease Rating Scale Part 4 | 3.0 units on a scale STANDARD_DEVIATION 0 | 5.3 units on a scale STANDARD_DEVIATION 3.81 | 4.5 units on a scale STANDARD_DEVIATION 4.04 | 4.6 units on a scale STANDARD_DEVIATION 3.98 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 3 Participants | 4 Participants | 7 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 1 Participants | 1 Participants | 2 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) White | 2 Participants | 30 Participants | 121 Participants | 153 Participants |
| Sex: Female, Male Female | 0 Participants | 6 Participants | 43 Participants | 49 Participants |
| Sex: Female, Male Male | 2 Participants | 28 Participants | 85 Participants | 115 Participants |
| Weight (kg) | 96.2 kg STANDARD_DEVIATION 0.64 | 85.0 kg STANDARD_DEVIATION 16.95 | 88.1 kg STANDARD_DEVIATION 22.6 | 87.6 kg STANDARD_DEVIATION 21.42 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 2 | 0 / 34 | 2 / 128 |
| other Total, other adverse events | 1 / 2 | 8 / 34 | 17 / 128 |
| serious Total, serious adverse events | 0 / 2 | 2 / 34 | 8 / 128 |
Outcome results
Primary
Change From Baseline in Montreal Cognitive Assessment (MoCA) Total Score.
MoCA is a 30-point, 1-page test designed to assess several cognitive domains, including visuospatial abilities (5 points), naming (3 points), attention (6 points), language (3 points), abstraction (2 points), delayed recall (5 points), and orientation to time and place (6 points). The total score ranges from 0 to 30, with higher scores indicating better performances.
Time frame: Baseline to Study Day 60
Population: Overall Number of Participants Analyzed represented by number analyzed at study visit Day 60. Safety Population: all subjects who sign informed consent form, complete baseline assessments, receive at least 1 dose of XADAGO. Safety population used to analyze all efficacy endpoints except MDS-UPDRS.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DA Switchers | Change From Baseline in Montreal Cognitive Assessment (MoCA) Total Score. | 0.0 score on a scale | Standard Deviation 1.41 |
| MAO-B Switchers | Change From Baseline in Montreal Cognitive Assessment (MoCA) Total Score. | 0.9 score on a scale | Standard Deviation 2.41 |
| MAO-B Naive | Change From Baseline in Montreal Cognitive Assessment (MoCA) Total Score. | 0.4 score on a scale | Standard Deviation 2.45 |
Primary
Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
MDS-UPDRS: 4-part assessment of the multiple clinical disabilities of Parkinson's Disease. Part I (13 items; Score 0-52) examines non-motor experiences, Part II (13 items; Score 0-52) examines motor experiences, Part III (33 items; Score 0-132) examines the cardinal motor disabilities and Part IV (6 items; Score 0-24) examines motor complications. Each Part has 0-4 ratings, where 0 (no problems) to 4 (severe problems) and scores for each part are summed to calculate the total score which ranges from 0-260. Higher scores represent worse outcomes for each part and total score.
Time frame: Baseline to Study Day 60
Population: Overall Number of Participants Analyzed represented by initial number analyzed at study visit Day 60; Number may differ for each Part (1-4) due to availability of score data. Evaluable Population: all subjects in the safety population who complete at least the MDS-UPDRS assessment at the Study Day 60 visit. Evaluable population will be used to analyze MDS-UPDRS endpoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| DA Switchers | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 1 | -5.0 score on a scale | Standard Deviation 5.66 |
| DA Switchers | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 2 | 0.0 score on a scale | Standard Deviation 2.83 |
| DA Switchers | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 3 | 1.5 score on a scale | Standard Deviation 2.12 |
| DA Switchers | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 4 | -0.5 score on a scale | Standard Deviation 3.54 |
| MAO-B Switchers | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 4 | -0.3 score on a scale | Standard Deviation 3.28 |
| MAO-B Switchers | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 1 | -1.0 score on a scale | Standard Deviation 5.13 |
| MAO-B Switchers | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 3 | -3.3 score on a scale | Standard Deviation 7.78 |
| MAO-B Switchers | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 2 | -2.3 score on a scale | Standard Deviation 5.4 |
| MAO-B Naive | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 4 | -0.2 score on a scale | Standard Deviation 3.05 |
| MAO-B Naive | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 2 | -2.6 score on a scale | Standard Deviation 5.28 |
| MAO-B Naive | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 3 | -4.5 score on a scale | Standard Deviation 11.1 |
| MAO-B Naive | Change From Baseline in Movement Disorders Society -Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Part 1 | -1.9 score on a scale | Standard Deviation 6.16 |
Primary
Change From Baseline in Parkinson's Disease Questionnaire (PDQ-39) Scores
PDQ-39 is a patient-reported outcome designed to address aspects of functioning and well-being for those affected by PD. Each of the 39 items is rated using a 5 point Likert scale with 0 for never having difficulties/problems and 4 for always having difficulties/problems. The sum score of the 39 items will be calculated and used for analysis, with scores ranging from 0-156. Higher scores indicate worse outcomes.
Time frame: Baseline to Study Day 60
Population: Overall Number of Participants Analyzed represented by number analyzed at study visit Day 60. Safety Population: all subjects who sign informed consent form, complete baseline assessments, receive at least 1 dose of XADAGO. Safety population used to analyze all efficacy endpoints except MDS-UPDRS.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DA Switchers | Change From Baseline in Parkinson's Disease Questionnaire (PDQ-39) Scores | -1.8 score on a scale | — |
| MAO-B Switchers | Change From Baseline in Parkinson's Disease Questionnaire (PDQ-39) Scores | 0.5 score on a scale | Standard Deviation 9.73 |
| MAO-B Naive | Change From Baseline in Parkinson's Disease Questionnaire (PDQ-39) Scores | -0.5 score on a scale | Standard Deviation 9.9 |
Primary
Clinical Global Impression of Change (CGI-C)
CGI-C is a 7-point scale depicting a Principal Investigator or certified Health Care Professional designee rating of the patient's overall improvement using a range of responses from a minimum of 1 (very much improved) to a maximum of 7 (very much worse).
Time frame: Study Day 60
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| DA Switchers | Clinical Global Impression of Change (CGI-C) | Minimally Worse | 0 Participants |
| DA Switchers | Clinical Global Impression of Change (CGI-C) | Very Much Worse | 0 Participants |
| DA Switchers | Clinical Global Impression of Change (CGI-C) | Much Worse | 0 Participants |
| DA Switchers | Clinical Global Impression of Change (CGI-C) | Minimally Improved | 0 Participants |
| DA Switchers | Clinical Global Impression of Change (CGI-C) | Very Much Improved | 0 Participants |
| DA Switchers | Clinical Global Impression of Change (CGI-C) | No Change | 1 Participants |
| DA Switchers | Clinical Global Impression of Change (CGI-C) | Much Improved | 1 Participants |
| MAO-B Switchers | Clinical Global Impression of Change (CGI-C) | Very Much Improved | 1 Participants |
| MAO-B Switchers | Clinical Global Impression of Change (CGI-C) | Minimally Worse | 2 Participants |
| MAO-B Switchers | Clinical Global Impression of Change (CGI-C) | No Change | 6 Participants |
| MAO-B Switchers | Clinical Global Impression of Change (CGI-C) | Much Worse | 1 Participants |
| MAO-B Switchers | Clinical Global Impression of Change (CGI-C) | Minimally Improved | 10 Participants |
| MAO-B Switchers | Clinical Global Impression of Change (CGI-C) | Very Much Worse | 0 Participants |
| MAO-B Switchers | Clinical Global Impression of Change (CGI-C) | Much Improved | 7 Participants |
| MAO-B Naive | Clinical Global Impression of Change (CGI-C) | Very Much Worse | 0 Participants |
| MAO-B Naive | Clinical Global Impression of Change (CGI-C) | Very Much Improved | 13 Participants |
| MAO-B Naive | Clinical Global Impression of Change (CGI-C) | Much Improved | 29 Participants |
| MAO-B Naive | Clinical Global Impression of Change (CGI-C) | Minimally Improved | 38 Participants |
| MAO-B Naive | Clinical Global Impression of Change (CGI-C) | No Change | 31 Participants |
| MAO-B Naive | Clinical Global Impression of Change (CGI-C) | Minimally Worse | 7 Participants |
| MAO-B Naive | Clinical Global Impression of Change (CGI-C) | Much Worse | 3 Participants |
Primary
Patient Global Impression of Change (PGI-C)
PGI-C is a 7-point scale depicting a patient's rating of overall improvement using a range of responses from 1 (very much improved) to 7 (very much worse).
Time frame: Study Day 60
Population: Participants Analyzed does not match the Participant Flow because some patient data was not captured for the visit. Safety Population: all subjects who sign informed consent form, complete baseline assessments, receive at least 1 dose of XADAGO. Safety population used to analyze all efficacy endpoints except MDS-UPDRS.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| DA Switchers | Patient Global Impression of Change (PGI-C) | Much Improved | 2 Participants |
| DA Switchers | Patient Global Impression of Change (PGI-C) | Minimally Improved | 0 Participants |
| DA Switchers | Patient Global Impression of Change (PGI-C) | Very Much Improved | 0 Participants |
| DA Switchers | Patient Global Impression of Change (PGI-C) | No Change | 0 Participants |
| DA Switchers | Patient Global Impression of Change (PGI-C) | Minimally Worse | 0 Participants |
| DA Switchers | Patient Global Impression of Change (PGI-C) | Much Worse | 0 Participants |
| DA Switchers | Patient Global Impression of Change (PGI-C) | Very Much Worse | 0 Participants |
| MAO-B Switchers | Patient Global Impression of Change (PGI-C) | Very Much Improved | 1 Participants |
| MAO-B Switchers | Patient Global Impression of Change (PGI-C) | Much Worse | 2 Participants |
| MAO-B Switchers | Patient Global Impression of Change (PGI-C) | No Change | 8 Participants |
| MAO-B Switchers | Patient Global Impression of Change (PGI-C) | Minimally Worse | 2 Participants |
| MAO-B Switchers | Patient Global Impression of Change (PGI-C) | Much Improved | 3 Participants |
| MAO-B Switchers | Patient Global Impression of Change (PGI-C) | Minimally Improved | 9 Participants |
| MAO-B Switchers | Patient Global Impression of Change (PGI-C) | Very Much Worse | 0 Participants |
| MAO-B Naive | Patient Global Impression of Change (PGI-C) | Minimally Worse | 2 Participants |
| MAO-B Naive | Patient Global Impression of Change (PGI-C) | Very Much Improved | 9 Participants |
| MAO-B Naive | Patient Global Impression of Change (PGI-C) | Minimally Improved | 31 Participants |
| MAO-B Naive | Patient Global Impression of Change (PGI-C) | Very Much Worse | 1 Participants |
| MAO-B Naive | Patient Global Impression of Change (PGI-C) | Much Improved | 25 Participants |
| MAO-B Naive | Patient Global Impression of Change (PGI-C) | No Change | 30 Participants |
| MAO-B Naive | Patient Global Impression of Change (PGI-C) | Much Worse | 2 Participants |
Primary
Treatment Satisfaction Questionnaire for Medication (TSQM-9) Scores
TSQM-9 consists of 9 questions to assess patients' satisfaction with medication using a range of responses from 1 (extremely dissatisfied) to (7 extremely satisfied). This patient reported outcome provides scores on three parts: effectiveness, convenience, and global satisfaction. The sum of the 9-questions will be calculated and used for analysis. The total score ranges from 0 to 63, with higher scores indicating better treatment satisfaction.
Time frame: Study Day 60
Population: Overall Number of Participants Analyzed represented by number analyzed at study visit Day 60. Safety Population: all subjects who sign informed consent form, complete baseline assessments, receive at least 1 dose of XADAGO. Safety population used to analyze all efficacy endpoints except MDS-UPDRS.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DA Switchers | Treatment Satisfaction Questionnaire for Medication (TSQM-9) Scores | 56.0 score on a scale | — |
| MAO-B Switchers | Treatment Satisfaction Questionnaire for Medication (TSQM-9) Scores | 38.9 score on a scale | Standard Deviation 7.73 |
| MAO-B Naive | Treatment Satisfaction Questionnaire for Medication (TSQM-9) Scores | 41.0 score on a scale | Standard Deviation 9.81 |