Trial Outcomes & Findings for Open Label Extension (OLE) Study of the Safety and Clinical Utility of IPX203 in Parkinson's Disease (PD) Participants With Motor Fluctuations (NCT NCT03877510)

Last Updated: 2023-07-13

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

419 participants

Primary outcome timeframe

From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])

Results posted on

2023-07-13

Participant Flow

Participants who successfully completed Study IPX203-B16-02 (NCT03670953) had the opportunity to enroll in the 9-month, open-label safety extension IPX203-B16-03 (NCT03877510) study. A total of 419 participants were enrolled.

Participants with Parkinson's Disease (PD) who were treated with stable regimens of carbidopa - levodopa (CD - LD) and had successfully completed previous IPX203-B16-02 study had the opportunity to enroll in this open-label study.

Participant milestones

Participant milestones
Measure	Open Label IPX203 All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Overall Study STARTED	419
Overall Study COMPLETED	352
Overall Study NOT COMPLETED	67

Reasons for withdrawal

Reasons for withdrawal
Measure	Open Label IPX203 All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Overall Study Death	6
Overall Study Lost to Follow-up	2
Overall Study Non-compliance with study drug	2
Overall Study Lack of Efficacy	14
Overall Study Adverse Event	20
Overall Study Withdrawal by Subject	22
Overall Study Miscellaneous	1

Baseline Characteristics

Open Label Extension (OLE) Study of the Safety and Clinical Utility of IPX203 in Parkinson's Disease (PD) Participants With Motor Fluctuations

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Open Label IPX203 n=419 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Age, Continuous	66.9 years STANDARD_DEVIATION 8.86 • n=5 Participants
Sex: Female, Male Female	140 Participants n=5 Participants
Sex: Female, Male Male	279 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	46 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	369 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	6 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants
Race (NIH/OMB) White	406 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])

Population: The Safety Analysis Set (SAS) included all participants who were treated with the open-label study drug in the study.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=419 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Number of Participants With Treatment-Emergent Adverse Events	221 Participants

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

Population: The Intent-to-Treat (ITT) Analysis Set included all participants who were treated with the open-label study drug in the study and had at least one post-baseline clinical utility assessment. "Overall Number of Participants Analyzed" included participants with available data at any timepoint. "Number Analyzed" included participants with available data at specified timepoint.

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).

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=412 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Total Month 3	56.5 score on a scale Standard Deviation 30.14
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Total Month 9/ET	59.0 score on a scale Standard Deviation 29.11
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Total Baseline	56.7 score on a scale Standard Deviation 28.64
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Total Month 6	56.0 score on a scale Standard Deviation 28.92

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=391 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Change From Baseline in Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Total	2.7 score on a scale Standard Deviation 16.44

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with available data at any timepoint. "Number Analyzed" included participants with available data at specified timepoint.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=412 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part I Baseline	9.9 score on a scale Standard Deviation 6.27
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part I Month 3	10.6 score on a scale Standard Deviation 6.43
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part I Month 6	10.7 score on a scale Standard Deviation 6.21
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part I Month 9/ET	11.0 score on a scale Standard Deviation 6.72

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=392 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Change From Baseline in Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part I	1.2 score on a scale Standard Deviation 4.56

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=412 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part II Baseline	12.8 score on a scale Standard Deviation 7.83
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part II Month 3	13.0 score on a scale Standard Deviation 7.96
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part II Month 6	12.8 score on a scale Standard Deviation 7.69
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part II Month 9/ET	13.6 score on a scale Standard Deviation 8.11

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=391 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Change From Baseline in Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part II	0.9 score on a scale Standard Deviation 4.81

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=412 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part III Baseline	27.5 score on a scale Standard Deviation 17.23
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part III Month 6	26.6 score on a scale Standard Deviation 17.50
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part III Month 9/ET	27.8 score on a scale Standard Deviation 16.79
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part III Month 3	26.8 score on a scale Standard Deviation 17.90

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=392 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Change From Baseline in Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part III	0.6 score on a scale Standard Deviation 11.29

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=412 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part IV Baseline	6.6 score on a scale Standard Deviation 2.96
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part IV Month 3	6.0 score on a scale Standard Deviation 3.15
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part IV Month 6	6.0 score on a scale Standard Deviation 3.08
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part IV Month 9/ET	6.6 score on a scale Standard Deviation 3.34

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=392 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Change From Baseline in Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Part IV	0.0 score on a scale Standard Deviation 2.64

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=412 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Sums of Part II and Part III Baseline	40.3 score on a scale Standard Deviation 23.09
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Sums of Part II and Part III Month 3	39.8 score on a scale Standard Deviation 24.15
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Sums of Part II and Part III Month 6	39.4 score on a scale Standard Deviation 23.24
Movement Disorders Society Version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Sums of Part II and Part III Month 9/ET	41.5 score on a scale Standard Deviation 22.96

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=391 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
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	1.5 score on a scale Standard Deviation 13.66

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

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.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=412 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Patient Global Impression of Severity (PGI-S) Month 3	3.8 score on a scale Standard Deviation 1.00
Patient Global Impression of Severity (PGI-S) Month 6	3.7 score on a scale Standard Deviation 0.99
Patient Global Impression of Severity (PGI-S) Baseline	3.7 score on a scale Standard Deviation 1.02
Patient Global Impression of Severity (PGI-S) Month 9/ET	3.8 score on a scale Standard Deviation 1.12

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=391 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Change From Baseline in Patient Global Impression of Severity (PGI-S)	0.0 score on a scale Standard Deviation 1.07

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

Population: ITT population with available data was analyzed.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=409 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Percentage of Participants With a PGI-S ≥ 4 and PGI-S ≥ 5 Month 9/ET PGI-S ≥ 4	65.4 Percentage of Participants
Percentage of Participants With a PGI-S ≥ 4 and PGI-S ≥ 5 Month 9/ET PGI-S ≥ 5	22.6 Percentage of Participants
Percentage of Participants With a PGI-S ≥ 4 and PGI-S ≥ 5 Baseline PGI-S ≥ 4	64.5 Percentage of Participants
Percentage of Participants With a PGI-S ≥ 4 and PGI-S ≥ 5 Baseline PGI-S ≥ 5	20.3 Percentage of Participants
Percentage of Participants With a PGI-S ≥ 4 and PGI-S ≥ 5 Month 3 PGI-S ≥ 4	66.7 Percentage of Participants
Percentage of Participants With a PGI-S ≥ 4 and PGI-S ≥ 5 Month 3 PGI-S ≥ 5	20.0 Percentage of Participants
Percentage of Participants With a PGI-S ≥ 4 and PGI-S ≥ 5 Month 6 PGI-S ≥ 4	62.5 Percentage of Participants
Percentage of Participants With a PGI-S ≥ 4 and PGI-S ≥ 5 Month 6 PGI-S ≥ 5	20.7 Percentage of Participants

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, Month 9/ET

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.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=412 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Clinical Global Impression of Severity (CGI-S) Baseline	3.8 score on a scale Standard Deviation 0.87
Clinical Global Impression of Severity (CGI-S) Month 3	3.8 score on a scale Standard Deviation 0.92
Clinical Global Impression of Severity (CGI-S) Month 6	3.7 score on a scale Standard Deviation 0.87
Clinical Global Impression of Severity (CGI-S) Month 9/ET	3.8 score on a scale Standard Deviation 0.97

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=390 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Change From Baseline in Clinical Global Impression of Severity (CGI-S)	0.0 score on a scale Standard Deviation 0.81

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

Population: ITT population with available data was analyzed.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=409 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Percentage of Participants With a CGI-S ≥ 4 and CGI-S ≥ 5 Baseline CGI-S ≥ 4	66.7 Percentage of participants
Percentage of Participants With a CGI-S ≥ 4 and CGI-S ≥ 5 Baseline CGI-S ≥ 5	19.6 Percentage of participants
Percentage of Participants With a CGI-S ≥ 4 and CGI-S ≥ 5 Month 3 CGI-S ≥ 4	64.8 Percentage of participants
Percentage of Participants With a CGI-S ≥ 4 and CGI-S ≥ 5 Month 3 CGI-S ≥ 5	18.3 Percentage of participants
Percentage of Participants With a CGI-S ≥ 4 and CGI-S ≥ 5 Month 6 CGI-S ≥ 4	61.2 Percentage of participants
Percentage of Participants With a CGI-S ≥ 4 and CGI-S ≥ 5 Month 6 CGI-S ≥ 5	14.0 Percentage of participants
Percentage of Participants With a CGI-S ≥ 4 and CGI-S ≥ 5 Month 9/ET CGI-S ≥ 4	65.3 Percentage of participants
Percentage of Participants With a CGI-S ≥ 4 and CGI-S ≥ 5 Month 9/ET CGI-S ≥ 5	17.6 Percentage of participants

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

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.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=411 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
39-item Parkinson's Disease Questionnaire (PDQ-39): Total Baseline	41.7 score on a scale Standard Deviation 27.71
39-item Parkinson's Disease Questionnaire (PDQ-39): Total Month 3	42.7 score on a scale Standard Deviation 27.73
39-item Parkinson's Disease Questionnaire (PDQ-39): Total Month 6	42.8 score on a scale Standard Deviation 28.15
39-item Parkinson's Disease Questionnaire (PDQ-39): Total Month 9/ET	45.1 score on a scale Standard Deviation 29.45

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=385 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Change From Baseline in PDQ-39: Total	4.4 score on a scale Standard Deviation 18.65

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

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.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=410 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Parkinson Anxiety Scale (PAS): Total Baseline	10.4 score on a scale Standard Deviation 8.18
Parkinson Anxiety Scale (PAS): Total Month 3	10.3 score on a scale Standard Deviation 8.20
Parkinson Anxiety Scale (PAS): Total Month 6	10.3 score on a scale Standard Deviation 7.67
Parkinson Anxiety Scale (PAS): Total Month 9/ET	11.1 score on a scale Standard Deviation 8.57

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=389 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Change From Baseline in Parkinson Anxiety Scale (PAS): Total	0.9 score on a scale Standard Deviation 6.41

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

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.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=412 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Non-Motor Symptom Assessment Scale (NMSS) for Parkinson's Disease (PD): Total Baseline	32.0 score on a scale Standard Deviation 28.95
Non-Motor Symptom Assessment Scale (NMSS) for Parkinson's Disease (PD): Total Month 3	32.5 score on a scale Standard Deviation 28.49
Non-Motor Symptom Assessment Scale (NMSS) for Parkinson's Disease (PD): Total Month 6	33.4 score on a scale Standard Deviation 29.19
Non-Motor Symptom Assessment Scale (NMSS) for Parkinson's Disease (PD): Total Month 9/ET	35.3 score on a scale Standard Deviation 30.08

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=389 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Change From Baseline in Non-Motor Symptom Assessment Scale (NMSS) for Parkinson's Disease (PD): Total	3.8 score on a scale Standard Deviation 22.33

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6, and Month 9/ET

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.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=412 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Parkinson's Disease Sleep Scale-2 (PDSS-2) Total Baseline	16.6 score on a scale Standard Deviation 9.72
Parkinson's Disease Sleep Scale-2 (PDSS-2) Total Month 3	15.8 score on a scale Standard Deviation 9.16
Parkinson's Disease Sleep Scale-2 (PDSS-2) Total Month 6	16.1 score on a scale Standard Deviation 9.29
Parkinson's Disease Sleep Scale-2 (PDSS-2) Total Month 9/ET	16.2 score on a scale Standard Deviation 9.28

SECONDARY outcome

Timeframe: Baseline, Month 9/ET

Population: ITT population. "Overall Number of Participants Analyzed" included participants with Baseline and Month 9/ET data.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=386 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Change From Baseline in Parkinson's Disease Sleep Scale-2 (PDSS-2):Total	-0.1 score on a scale Standard Deviation 8.92

SECONDARY outcome

Timeframe: Month 3, Month 6 and Month 9/ET

Population: ITT population with available data was analyzed.

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.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=392 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Treatment Satisfaction Assessment (TSA) Month 3	5.2 score on a scale Standard Deviation 1.25
Treatment Satisfaction Assessment (TSA) Month 6	5.4 score on a scale Standard Deviation 1.19
Treatment Satisfaction Assessment (TSA) Month 9/ET	5.2 score on a scale Standard Deviation 1.39

SECONDARY outcome

Timeframe: Month 3, Month 6, and Month 9/ET

Population: ITT population with available data was analyzed.

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.

Outcome measures

Outcome measures
Measure	Open Label IPX203 n=392 Participants All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Percentage of Participants With TSA Scores 5-7 (Satisfied) Versus Scores 1-4 (Dissatisfied or Neutral) Month 3: Percentage of TSA Scores 1-4 (Dissatisfied or Neutral)	19.6 Percentage of Participants
Percentage of Participants With TSA Scores 5-7 (Satisfied) Versus Scores 1-4 (Dissatisfied or Neutral) Month 3: Percentage of TSA Scores 5-7 (Satisfied)	80.4 Percentage of Participants
Percentage of Participants With TSA Scores 5-7 (Satisfied) Versus Scores 1-4 (Dissatisfied or Neutral) Month 6: Percentage of TSA Scores 1-4 (Dissatisfied or Neutral)	15.6 Percentage of Participants
Percentage of Participants With TSA Scores 5-7 (Satisfied) Versus Scores 1-4 (Dissatisfied or Neutral) Month 6: Percentage of TSA Scores 5-7 (Satisfied)	84.4 Percentage of Participants
Percentage of Participants With TSA Scores 5-7 (Satisfied) Versus Scores 1-4 (Dissatisfied or Neutral) Month 9/ET: Percentage of TSA Scores 1-4 (Dissatisfied or Neutral)	23.7 Percentage of Participants
Percentage of Participants With TSA Scores 5-7 (Satisfied) Versus Scores 1-4 (Dissatisfied or Neutral) Month 9/ET: Percentage of TSA Scores 5-7 (Satisfied)	76.3 Percentage of Participants

Adverse Events

Open Label IPX203

Serious events: 42 serious events

Other events: 99 other events

Deaths: 6 deaths

Serious adverse events

Serious adverse events
Measure	Open Label IPX203 n=419 participants at risk All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Blood and lymphatic system disorders Anemia	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Cardiac disorders Acute myocardial infarction	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Cardiac disorders Atrial fibrillation	0.48% 2/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Cardiac disorders Cardiac failure	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Cardiac disorders Cardiogenic shock	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Ear and labyrinth disorders Vertigo	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Gastrointestinal disorders Abdominal distension	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Gastrointestinal disorders Colitis	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Gastrointestinal disorders Volvulus	0.48% 2/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Gastrointestinal disorders Vomiting	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
General disorders Asthenia	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
General disorders Chest pain	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
General disorders Drowning	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
General disorders Non-cardiac chest pain	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
General disorders Pyrexia	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Immune system disorders Hypersensitivity	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Infections and infestations COVID-19	0.72% 3/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Infections and infestations COVID-19 pneumonia	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Infections and infestations Encephalitis	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Infections and infestations Gastroenteritis viral	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Infections and infestations Gastrointestinal infection	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Infections and infestations Herpes zoster	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Infections and infestations Pneumonia	0.48% 2/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Infections and infestations Post procedural infection	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Infections and infestations Sepsis	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Injury, poisoning and procedural complications Concussion	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Injury, poisoning and procedural complications Fall	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Injury, poisoning and procedural complications Femur fracture	0.48% 2/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Injury, poisoning and procedural complications Head injury	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Injury, poisoning and procedural complications Joint dislocation	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Injury, poisoning and procedural complications Overdose	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Injury, poisoning and procedural complications Spinal cord injury cervical	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Metabolism and nutrition disorders Dehydration	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Metabolism and nutrition disorders Hypokalaemia	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Musculoskeletal and connective tissue disorders Lumbar spinal stenosis	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Musculoskeletal and connective tissue disorders Scoliosis	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Lung neoplasm malignant	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Nervous system disorders Cauda equina syndrome	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Nervous system disorders Cerebrovascular accident	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Nervous system disorders Epilepsy	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Nervous system disorders Freezing phenomenon	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Nervous system disorders Parkinson's disease	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Nervous system disorders Peripheral sensory neuropathy	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Nervous system disorders Polyneuropathy	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Nervous system disorders Seizure	0.72% 3/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Nervous system disorders Subarachnoid haemorrhage	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Nervous system disorders Syncope	0.48% 2/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Psychiatric disorders Confusional state	0.72% 3/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Psychiatric disorders Delirium	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Psychiatric disorders Depression	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Psychiatric disorders Hypersexuality	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Psychiatric disorders Mental status changes	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Psychiatric disorders Psychotic disorder	0.48% 2/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Renal and urinary disorders Nephrolithiasis	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Renal and urinary disorders Neurogenic bladder	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Respiratory, thoracic and mediastinal disorders Aspiration	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	0.48% 2/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Respiratory, thoracic and mediastinal disorders Hypoxia	0.24% 1/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.48% 2/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])

Other adverse events

Other adverse events
Measure	Open Label IPX203 n=419 participants at risk All participants received oral administration of extended-release capsules IPX203 dosing regimen that was determined at the end of the IPX203 dose conversion period of Study IPX203-B16-02 (NCT03670953) for nine months of open-label therapy. The dose and dosing frequency was determined by the investigator to achieve the optimal balance of efficacy and safety.
Gastrointestinal disorders Constipation	2.6% 11/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Infections and infestations COVID-19	2.4% 10/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Infections and infestations Urinary tract infection	5.0% 21/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Injury, poisoning and procedural complications Fall	5.0% 21/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Musculoskeletal and connective tissue disorders Back pain	3.6% 15/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])
Nervous system disorders Dyskinesia	5.0% 21/419 • From first dose up to 1 day after last dose (Up to 9 months/Early Termination [ET])

Additional Information

Pamela Fitzpatrick, Senior Director, Specialty Regulatory Affairs

Impax Laboratories, LLC

Phone: 631-633-2104

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place