Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Number of Participants With Treatment-Emergent Adverse Events |
An adverse event (AE) is any untoward medical occurrence in a participant or clinical trial participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (e.g., an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An AE was considered as treatment-emergent if the date of onset was on or after the date of the first open-label study drug administration in this Study IPX203-B16-03 and no later than 1 day after the last study drug dose in the study. |
From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET]) |
|
| Secondary |
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Total |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Change From Baseline in Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Total |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). |
Baseline, Month 9/ET |
|
| Secondary |
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part I |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). Part I score ranges from 0 to 52. A higher score indicated more severe symptoms of PD. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Change From Baseline in Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part I |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). Part I score ranges from 0 to 52. A higher score indicated more severe symptoms of PD. |
Baseline, Month 9/ET |
|
| Secondary |
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part II |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). Part II score ranges from 0 to 52. A higher score indicated more severe symptoms of PD. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Change From Baseline in Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part II |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). Part II score ranges from 0 to 52. A higher score indicated more severe symptoms of PD. |
Baseline, Month 9/ET |
|
| Secondary |
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part III |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). Part III score ranges from 0 to 132. A higher score indicated more severe symptoms of PD. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Change From Baseline in Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part III |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). Part III score ranges from 0 to 132. A higher score indicated more severe symptoms of PD. |
Baseline, Month 9/ET |
|
| Secondary |
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part IV |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). Part IV score ranges from 0 to 24. A higher score indicated more severe symptoms of PD. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Change From Baseline in Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part IV |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). Part IV score ranges from 0 to 24. A higher score indicated more severe symptoms of PD. |
Baseline, Month 9/ET |
|
| Secondary |
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Sums of Part II and Part III |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). The scale range for Part II+III score is 0-184. A higher score indicated more severe symptoms of PD. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Change From Baseline in Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Sums of Part II and Part III |
MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. (Part I; 13 items) non-motor experiences of daily living, (Part II; 13 items) motor experiences of daily living completed by the participants, (Part III; 33 items) motor examination of PD and was administered by the rater, and (Part IV; 6 items) motor complication integrates participant-derived information with the rater's clinical observations and judgements and is completed by the rater. For each question a numeric score was assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Total Score equals the sum of Parts I, II, and III and IV (Range 0-260). The scale range for Part II+III score is 0-184. A higher score indicated more severe symptoms of PD. |
Baseline, Month 9/ET |
|
| Secondary |
Patient Global Impression of Severity (PGI-S) |
The PGI-S is a participant answered assessment rating Parkinson's disease severity on a scale of 1 to 7; 1-Normal, not at all ill, 2-Borderline ill, 3-Mildly ill, 4-Moderately ill, 5-Markedly ill, 6-Severely ill, 7-Extremely severely ill. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Change From Baseline in Patient Global Impression of Severity (PGI-S) |
The PGI-S is a participant answered assessment rating Parkinson's disease severity on a scale of 1 to 7; 1-Normal, not at all ill, 2-Borderline ill, 3-Mildly ill, 4-Moderately ill, 5-Markedly ill, 6-Severely ill, 7-Extremely severely ill. |
Baseline, Month 9/ET |
|
| Secondary |
Percentage of Participants With a PGI-S = 4 and PGI-S = 5 |
The PGI-S is a participant answered assessment rating Parkinson's disease severity on a scale of 1 to 7; 1-Normal, not at all ill, 2-Borderline ill, 3-Mildly ill, 4-Moderately ill, 5-Markedly ill, 6-Severely ill, 7-Extremely severely ill. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Clinical Global Impression of Severity (CGI-S) |
The CGI-S is a clinician scale rating the severity of the participant's illness on a scale of 1 to 7; 1-Normal, not at all ill, 2-Borderline ill, 3-Mildly ill, 4-Moderately ill, 5-Markedly ill, 6-Severely ill, 7-Among the most extremely ill of participants. |
Baseline, Month 3, Month 6, Month 9/ET |
|
| Secondary |
Change From Baseline in Clinical Global Impression of Severity (CGI-S) |
The CGI-S is a clinician scale rating the severity of the participant's illness on a scale of 1 to 7; 1-Normal, not at all ill, 2-Borderline ill, 3-Mildly ill, 4-Moderately ill, 5-Markedly ill, 6-Severely ill, 7-Among the most extremely ill of participants. |
Baseline, Month 9/ET |
|
| Secondary |
Percentage of Participants With a CGI-S = 4 and CGI-S = 5 |
The CGI-S is a clinician scale rating the severity of the participant's illness on a scale of 1 to 7; 1-Normal, not at all ill, 2-Borderline ill, 3-Mildly ill, 4-Moderately ill, 5-Markedly ill, 6-Severely ill, 7-Among the most extremely ill of participants. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
39-item Parkinson's Disease Questionnaire (PDQ-39): Total |
The PDQ-39 is a self-reported outcome of 39 questions relating to 8 domains: mobility (Questions 1-10), activities of daily living (ADL) (Questions 11-16), emotional well-being (Questions 17-22), stigma (Questions 23-26), social support (Questions 27-29), cognition (Questions 30-33), communication (Questions 34-36) and bodily discomfort (Questions 37-39). Each question is answered on a 5-point scale from 0 (Never) to 4 (Always / Cannot Do At All). Scores are calculated by summing the answers to the questions in the domain and converting to a scale from 0 to 100. Higher scores are associated with the more severe symptoms of the disease such as tremor and stiffness. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Change From Baseline in PDQ-39: Total |
The PDQ-39 is a self-reported outcome of 39 questions relating to 8 domains: mobility (Questions 1-10), activities of daily living (ADL) (Questions 11-16), emotional well-being (Questions 17-22), stigma (Questions 23-26), social support (Questions 27-29), cognition (Questions 30-33), communication (Questions 34-36) and bodily discomfort (Questions 37-39). Each question is answered on a 5-point scale from 0 (Never) to 4 (Always / Cannot Do At All). Scores are calculated by summing the answers to the questions in the domain and converting to a scale from 0 to 100. Higher scores are associated with the more severe symptoms of the disease such as tremor and stiffness. |
Baseline, Month 9/ET |
|
| Secondary |
Parkinson Anxiety Scale (PAS): Total |
The PAS is a three part participant answered assessment. Part 1 has 5 questions measuring persistent anxiety. Each question ranges from 0 - not at all, or never to 4 severe, or nearly always. Best score is 0; worst score is 20. Part 2 has 4 questions measuring episodic anxiety. Each question ranges from 0 - never to 4 - nearly always. Best score is 0; worst score is 16. Part 3 has 3 questions measuring avoidance behavior. Each question ranges from 0, never to 4 nearly always. Best score is 0; worst score is 12. Totals for all three parts are summed and ranges from 0 to 48, higher scores are associated with the more severe symptoms. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Change From Baseline in Parkinson Anxiety Scale (PAS): Total |
The PAS is a three part participant answered assessment. Part 1 has 5 questions measuring persistent anxiety. Each question ranges from 0 - not at all, or never to 4 severe, or nearly always. Best score is 0; worst score is 20. Part 2 has 4 questions measuring episodic anxiety. Each question ranges from 0 - never to 4 - nearly always. Best score is 0; worst score is 16. Part 3 has 3 questions measuring avoidance behavior. Each question ranges from 0, never to 4 nearly always. Best score is 0; worst score is 12. Totals for all three parts are summed and ranges from 0 to 48, higher scores are associated with the more severe symptoms. |
Baseline, Month 9/ET |
|
| Secondary |
Non-Motor Symptom Assessment Scale (NMSS) for Parkinson's Disease (PD): Total |
The NMSS assesses non-motor symptoms in Parkinson's Disease. It consists of 30 questions grouped into 9 domains: cardiovascular, sleep/fatigue, mood/cognition, perceptual problems/hallucinations, attention/memory, gastro-intestinal tract, urinary, sexual function, and miscellaneous. Severity symptoms are rated on a scale of 0-3 with 3 being most severe; and frequency on a scale of 1-4 with 4 being most frequent. Each question is answered with a severity and frequency rating which are then multiplied. The sum of the products gives the total score which ranges from 0 to 360 with a lower score more desirable than a higher score. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Change From Baseline in Non-Motor Symptom Assessment Scale (NMSS) for Parkinson's Disease (PD): Total |
The NMSS assesses non-motor symptoms in Parkinson's Disease. It consists of 30 questions grouped into 9 domains: cardiovascular, sleep/fatigue, mood/cognition, perceptual problems/hallucinations, attention/memory, gastro-intestinal tract, urinary, sexual function, and miscellaneous. Severity symptoms are rated on a scale of 0-3 with 3 being most severe; and frequency on a scale of 1-4 with 4 being most frequent. Each question is answered with a severity and frequency rating which are then multiplied. The sum of the products gives the total score which ranges from 0 to 360 with a lower score more desirable than a higher score. |
Baseline, Month 9/ET |
|
| Secondary |
Parkinson's Disease Sleep Scale-2 (PDSS-2) Total |
The PDSS-2 is a 15 question participant response scale measuring the severity of sleep disturbance. Three domains are defined: disturbed sleep (Questions 1-3, 8, 14), motor symptoms at night (Questions 4-6, 12, 13), PD symptoms at night (Questions 7, 9-11, 15). Each question is rated as very often to never on a scale of 0-4. The best overall score is 0; the worst overall score is 60. |
Baseline, Month 3, Month 6, and Month 9/ET |
|
| Secondary |
Change From Baseline in Parkinson's Disease Sleep Scale-2 (PDSS-2):Total |
The PDSS-2 is a 15 question participant response scale measuring the severity of sleep disturbance. Three domains are defined: disturbed sleep (Questions 1-3, 8, 14), motor symptoms at night (Questions 4-6, 12, 13), PD symptoms at night (Questions 7, 9-11, 15). Each question is rated as very often to never on a scale of 0-4. The best overall score is 0; the worst overall score is 60. |
Baseline, Month 9/ET |
|
| Secondary |
Treatment Satisfaction Assessment (TSA) |
The TSA is a participant answered assessment rating treatment satisfaction on a scale of 1 to 7; 1 = Very much dissatisfied being least satisfied and 7 = Very much satisfied. |
Month 3, Month 6 and Month 9/ET |
|
| Secondary |
Percentage of Participants With TSA Scores 5-7 (Satisfied) Versus Scores 1-4 (Dissatisfied or Neutral) |
The TSA is a participant answered assessment rating treatment satisfaction on a scale of 1 to 7; 1 = Very much dissatisfied being least satisfied and 7 = Very much satisfied. |
Month 3, Month 6, and Month 9/ET |
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