Trial Outcomes & Findings for Safety and Biomarker Study of PTC-589 in Participants With Parkinson's Disease (NCT NCT02462603)
NCT ID: NCT02462603
Last Updated: 2022-05-03
Results Overview
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
COMPLETED
PHASE2
44 participants
Baseline up to 30 days after last dose of study drug (up to 4 months)
2022-05-03
Participant Flow
Participant milestones
| Measure |
PTC589
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 milligrams (mg) (2 tablets of 250 mg each) orally twice daily (BID) for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Overall Study
STARTED
|
44
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
41
|
|
Overall Study
EITT Population
|
40
|
|
Overall Study
COMPLETED
|
40
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
PTC589
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 milligrams (mg) (2 tablets of 250 mg each) orally twice daily (BID) for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Overall Study
Investigator Decision
|
1
|
|
Overall Study
Non-Compliance
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
Safety and Biomarker Study of PTC-589 in Participants With Parkinson's Disease
Baseline characteristics by cohort
| Measure |
PTC589
n=41 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Age, Continuous
|
59.2 years
STANDARD_DEVIATION 8.21 • n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
33 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
39 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
40 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline up to 30 days after last dose of study drug (up to 4 months)Population: Safety population included any participant who received at least 1 dose of PTC589.
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Outcome measures
| Measure |
PTC589
n=41 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Number of Participants With Drug-Related Serious Adverse Events (SAEs)
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, Month 3Population: Efficacy intent-to-treat (EITT) population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment.
The MDS-UPDRS is a tool for monitoring the impact of Parkinson's disease, the degree of disability caused, and complications from treatment. Part I (13 items) evaluates nonmotor experiences of daily living (nM-EDL); Part II (13 items) evaluates motor experiences of daily living (M-EDL; Part III (18 items) is a motor examination; Part IV (6 items) examines motor complications (for example, motor fluctuations and dyskinesias). Each item was rated on a 5-point scale, ranging from 0 (normal) to 4 (severe), with higher score indicating greater severity and more impairment. Total score for Part I (nM-EDL) and Part II (M-EDL) each ranges from 0-52; for Part III (motor examination) ranges from 0-72; and for Part IV (motor complications) ranges from 0-24; with higher scores in each range for all 4 parts reflecting greater severity.
Outcome measures
| Measure |
PTC589
n=40 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3
M-EDL Total Score: Baseline
|
5.8 units on a scale
Standard Deviation 4.13
|
|
Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3
M-EDL Total Score: Change at Month 3
|
0.1 units on a scale
Standard Deviation 3.54
|
|
Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3
Motor Examination Total Score: Baseline
|
22.5 units on a scale
Standard Deviation 8.60
|
|
Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3
Motor Examination Total Score: Change at Month 3
|
-0.6 units on a scale
Standard Deviation 6.72
|
|
Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3
Motor Complications Total Score: Baseline
|
1.3 units on a scale
Standard Deviation 2.34
|
|
Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3
Motor Complications Total Score: Change at Month 3
|
-0.1 units on a scale
Standard Deviation 2.17
|
|
Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3
nM-EDL Total Score: Baseline
|
5.0 units on a scale
Standard Deviation 4.51
|
|
Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3
nM-EDL Total Score: Change at Month 3
|
-0.1 units on a scale
Standard Deviation 3.93
|
SECONDARY outcome
Timeframe: Baseline, Month 3Population: EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment.
Non-motor symptoms were evaluated using the NMSS which was divided into 30 questions in 9 different domains including such symptoms as dribbling saliva, constipation, depression, sleep disorders, apathy, hallucinations and dementia. Symptoms were quantified based on their severity (using a scale of 0 \[none\] to 3 \[severe\]) and frequency (using a scale of 0 \[rarely\] to 4 \[very frequent\]). Total score derived from adding up the product of the frequency score times severity score for each of the 30 questions. Total score ranged from 0 to 360, with a lower score indicating fewer symptoms.
Outcome measures
| Measure |
PTC589
n=40 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Change From Baseline in Non-motor Symptoms Scale (NMSS) Total Score at Month 3
Baseline
|
15.7 units on a scale
Standard Deviation 14.84
|
|
Change From Baseline in Non-motor Symptoms Scale (NMSS) Total Score at Month 3
Change at Month 3
|
-0.6 units on a scale
Standard Deviation 14.79
|
SECONDARY outcome
Timeframe: Baseline, Month 3Population: EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment.
The PDQ-39 is a self-administered questionnaire for participants with Parkinson's disease that has 39 questions grouped in 8 dimensions: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Each item was scored on a 5-point Likert scale (0 to 4) to indicate the frequency of each event; 0 = never, 1 = occasionally, 2 = sometimes, 3 = often, and 4 = always or cannot do at all. Each dimension's total score ranged from 0-100, with lower scores indicating better health, and higher scores indicating more severe symptoms.
Outcome measures
| Measure |
PTC589
n=40 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Mobility: Baseline
|
7.06 units on a scale
Standard Deviation 12.543
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Mobility: Change at Month 3
|
0.19 units on a scale
Standard Deviation 7.281
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Activities of daily living: Baseline
|
11.56 units on a scale
Standard Deviation 10.096
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Activities of daily living: Change at Month 3
|
0.21 units on a scale
Standard Deviation 10.676
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Emotional well-being: Baseline
|
10.84 units on a scale
Standard Deviation 11.387
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Emotional well-being: Change at Month 3
|
0.10 units on a scale
Standard Deviation 10.180
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Stigma: Baseline
|
15.96 units on a scale
Standard Deviation 17.685
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Stigma: Change at Month 3
|
-0.94 units on a scale
Standard Deviation 16.602
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Social support: Baseline
|
1.88 units on a scale
Standard Deviation 4.811
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Social support: Change at Month 3
|
3.12 units on a scale
Standard Deviation 9.750
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Cognition: Baseline
|
9.70 units on a scale
Standard Deviation 12.085
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Cognition: Change at Month 3
|
-1.24 units on a scale
Standard Deviation 8.747
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Communication: Baseline
|
6.25 units on a scale
Standard Deviation 9.388
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Communication: Change at Month 3
|
1.25 units on a scale
Standard Deviation 10.777
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Bodily discomfort: Baseline
|
16.87 units on a scale
Standard Deviation 15.390
|
|
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Bodily discomfort: Change at Month 3
|
0.01 units on a scale
Standard Deviation 14.501
|
SECONDARY outcome
Timeframe: Baseline, Month 3Population: EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment.
EQ-5D is a questionnaire designed to provide measures of health-related quality of life states, consisting of 5 domains (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension has a 3 point response scale designed to indicate the level of the problem: 1 = no problems, 2 = some problems, 3 = extreme problems. A higher score indicated an increase in the level of problem. The EQ-5D also contains a visual analog scale (EQ-VAS), which records the respondent's self-rated health status on a vertical graduated visual analog scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state). Higher score indicated improvement.
Outcome measures
| Measure |
PTC589
n=40 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
Mobility: Baseline
|
1.2 units on a scale
Standard Deviation 0.36
|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
Mobility: Change at Month 3
|
0 units on a scale
Standard Deviation 0.36
|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
Personal Care: Baseline
|
1.1 units on a scale
Standard Deviation 0.27
|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
Personal Care: Change at Month 3
|
0 units on a scale
Standard Deviation 0.36
|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
Usual Activities: Baseline
|
1.2 units on a scale
Standard Deviation 0.42
|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
Usual Activities: Change at Month 3
|
0.1 units on a scale
Standard Deviation 0.35
|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
Pain/Discomfort: Baseline
|
1.3 units on a scale
Standard Deviation 0.47
|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
Pain/Discomfort: Change at Month 3
|
0.2 units on a scale
Standard Deviation 0.48
|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
Anxiety/Depression: Baseline
|
1.1 units on a scale
Standard Deviation 0.27
|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
Anxiety/Depression: Change at Month 3
|
0.2 units on a scale
Standard Deviation 0.50
|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
VAS Score: Baseline
|
81.0 units on a scale
Standard Deviation 12.00
|
|
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
VAS Score: Change at Month 3
|
-1.9 units on a scale
Standard Deviation 8.68
|
SECONDARY outcome
Timeframe: Month 3Population: EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment. Here, 'Number analyzed' signifies participants with normal, mild, moderate, or severe cognition impairment. Data for a specific severity level was not collected if no evaluable participants were available.
MoCA is a 30-point questionnaire for cognitive dysfunction. It assesses different cognitive domains: attention and concentration, executive functions, memory, language, visuoconstructional skills, conceptual thinking, calculations, and orientation. Scores on the MoCA range from 0-30 with 26-30 indicating normal global cognition; 18-25 mild cognitive impairment; 10-17 moderate cognitive impairment; and \<10 severe cognitive impairment.
Outcome measures
| Measure |
PTC589
n=40 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Montreal Cognitive Assessment (MoCA) Score
Normal global cognition
|
28.5 units on a scale
Standard Deviation 1.25
|
|
Montreal Cognitive Assessment (MoCA) Score
Mild cognitive impairment
|
23.4 units on a scale
Standard Deviation 1.40
|
SECONDARY outcome
Timeframe: Month 3Population: EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of Month 3 assessment. 'Number analyzed' = participants with symptom score \>1 for normal, mild, borderline, moderate, severe, or extreme depression. Data for a specific severity level was not collected if no evaluable participants were available.
The BDI is a self-reporting 21-item scoring tool that measures characteristic attitudes and symptoms of depression, including physical symptoms. Each of the 21 items on BDI tool represent a depressive symptom. The symptoms are each scored on a 4-point Likert scale of 0 (symptom is absent) to 3 (symptom is severe). Scores for each symptom are added up to obtain the total scores for all 21 items, which are interpreted as follows 1-10 (normal); 11-16 (mild mood disturbance); 17-20 (borderline clinical depression); 21-30 (moderate depression); 31-40 (severe depression); and \>40 (extreme depression). Participants with symptom score of 0 were not included in the summary.
Outcome measures
| Measure |
PTC589
n=40 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Beck Depression Inventory (BDI) Score
Normal
|
3.6 units on a scale
Standard Deviation 2.41
|
|
Beck Depression Inventory (BDI) Score
Mild mood disturbance
|
13.1 units on a scale
Standard Deviation 1.83
|
SECONDARY outcome
Timeframe: Baseline, Month 3Population: EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment.
The MADRS is a clinician-rated tool for measuring changes in depressive symptom severity. Ten core symptoms and cognitive features (feelings of sadness, lassitude, pessimism, inner tension, suicidality, reduced sleep or appetite, difficulty concentrating, and a lack of interest) were rated on a severity scale of 0 (no symptoms)) to 6 (symptoms of maximum severity). The total score was the sum of the scores on the 10 items, ranging from 0 to 60 with a higher score indicating increasing depressive symptoms.
Outcome measures
| Measure |
PTC589
n=40 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Change From Baseline in Montgomery and Asberg Depression Rating Scale (MADRS) Total Score at Month 3
Baseline
|
2.4 units on a scale
Standard Deviation 2.60
|
|
Change From Baseline in Montgomery and Asberg Depression Rating Scale (MADRS) Total Score at Month 3
Change at Month 3
|
0.1 units on a scale
Standard Deviation 3.00
|
SECONDARY outcome
Timeframe: Baseline, Month 3Population: EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
Timed motor tests are simple, objective, quantitative measures for the assessment of Parkinson's disease. They include, in on-medication and off-medication state, timed recorded physical movements. Time Up and Go Test (TUG) is one of timed motor tests which is used to assess a person's mobility and requires both static and dynamic balance. This is a walking assessment. Participants start in the seated position, stand up, walk 7 meters, turn around, and sit back down. The entire process from leaving the chair to returning to the chair was timed. The total time was summarized under ON state with participants on dopamine therapy.
Outcome measures
| Measure |
PTC589
n=18 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Change From Baseline in Time to Complete Time Up and Go (TUG) Test in ON State at Month 3
Baseline
|
8.637 seconds
Standard Deviation 1.8763
|
|
Change From Baseline in Time to Complete Time Up and Go (TUG) Test in ON State at Month 3
Change at Month 3
|
-0.347 seconds
Standard Deviation 1.1056
|
SECONDARY outcome
Timeframe: 0 hour (predose) and 0.5, 1, 2, 4, 6, 8, and 12 hours postdose at Month 1 and 3Population: EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
Outcome measures
| Measure |
PTC589
n=23 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Maximum Observed Plasma Concentration (Cmax) of PTC589
Month 1
|
3718.3 nanograms (ng)/milliliter (mL)
Standard Error 311.6
|
|
Maximum Observed Plasma Concentration (Cmax) of PTC589
Month 3
|
2903.5 nanograms (ng)/milliliter (mL)
Standard Error 249.0
|
SECONDARY outcome
Timeframe: Month 3Population: EITT population included any participant who received at least 1 dose of EPI-589.
Glutathione lowest limit of quantification (LLOQ) = 0.01 micromoles (uM) and upper limit of quantification (ULOQ) = 27.83 uM in plasma.
Outcome measures
| Measure |
PTC589
n=40 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Level of Disease-Related Biomarker (Glutathione) in Plasma
|
1.54 µM
Interval 0.37 to 8.33
|
SECONDARY outcome
Timeframe: Month 3Population: EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
Glutathione LLOQ = 0.002 uM and ULOQ = 0.35 uM in CSF.
Outcome measures
| Measure |
PTC589
n=36 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF)
|
0.10 µM
Interval 0.04 to 0.23
|
SECONDARY outcome
Timeframe: Month 3Population: EITT population included any participant who received at least 1 dose of EPI-589.
Glutathione LLOQ = 0.01 uM, and ULOQ = 1.39 uM in urine.
Outcome measures
| Measure |
PTC589
n=40 Participants
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Level of Disease-Related Biomarker (Glutathione) in Urine
|
0.0000061 µM
Interval 0.00000034 to 0.000033
|
Adverse Events
PTC589
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
PTC589
n=41 participants at risk
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.
|
|---|---|
|
Gastrointestinal disorders
Nausea
|
17.1%
7/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Gastrointestinal disorders
Vomiting
|
9.8%
4/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
General disorders
Fatigue
|
7.3%
3/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Injury, poisoning and procedural complications
Post lumbar puncture syndrome
|
7.3%
3/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.2%
5/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.3%
3/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Nervous system disorders
Dizziness
|
9.8%
4/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Nervous system disorders
Headache
|
19.5%
8/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Eye disorders
Abnormal sensation in eye
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Eye disorders
Dry eye
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Eye disorders
Eye pain
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Eye disorders
Ocular discomfort
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Eye disorders
Ocular hyperaemia
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Gastrointestinal disorders
Diarrhoea
|
4.9%
2/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Gastrointestinal disorders
Dyspepsia
|
4.9%
2/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Gastrointestinal disorders
Eructation
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Gastrointestinal disorders
Flatulence
|
4.9%
2/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Gastrointestinal disorders
Salivary hypersecretion
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
General disorders
Asthenia
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
General disorders
Malaise
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
General disorders
Pain
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
General disorders
Pyrexia
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Infections and infestations
Influenza
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Infections and infestations
Localised infection
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Infections and infestations
Respiratory tract infection viral
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Infections and infestations
Skin infection
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Infections and infestations
Upper respiratory tract infection
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Injury, poisoning and procedural complications
Facial bones fracture
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Injury, poisoning and procedural complications
Fall
|
4.9%
2/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Injury, poisoning and procedural complications
Muscle strain
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Investigations
Aspartate aminotransferase increased
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Investigations
Blood cholesterol increased
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Investigations
Low density lipoprotein increased
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Metabolism and nutrition disorders
Diabetes mellitus
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Metabolism and nutrition disorders
Gout
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
4.9%
2/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
4.9%
2/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
4.9%
2/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Musculoskeletal and connective tissue disorders
Plantar fasciitis
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Musculoskeletal and connective tissue disorders
Tendonitis
|
4.9%
2/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Nervous system disorders
Bradykinesia
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Nervous system disorders
Dyskinesia
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Nervous system disorders
Dystonia
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Nervous system disorders
Hypoaesthesia
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Nervous system disorders
Hypogeusia
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Nervous system disorders
Peroneal nerve palsy
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Nervous system disorders
Tremor
|
4.9%
2/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Psychiatric disorders
Depressed mood
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Psychiatric disorders
Depression
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Psychiatric disorders
Insomnia
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Psychiatric disorders
Nightmare
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Renal and urinary disorders
Pollakiuria
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
4.9%
2/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal inflammation
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
2.4%
1/41 • Baseline up to 30 days after last dose of study drug (up to 4 months)
Safety population included any participant who received at least 1 dose of PTC589.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER