Trial Outcomes & Findings for Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211) (NCT NCT00931944)
NCT ID: NCT00931944
Last Updated: 2021-08-16
Results Overview
Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
74 participants
Primary outcome timeframe
180 weeks
Results posted on
2021-08-16
Participant Flow
Participant milestones
| Measure |
300 mg Per Day
KNS-760704 (dexpramipexole) - 150 mg BID
|
|---|---|
|
Overall Study
STARTED
|
74
|
|
Overall Study
COMPLETED
|
19
|
|
Overall Study
NOT COMPLETED
|
55
|
Reasons for withdrawal
| Measure |
300 mg Per Day
KNS-760704 (dexpramipexole) - 150 mg BID
|
|---|---|
|
Overall Study
Adverse Event
|
4
|
|
Overall Study
W/D Consent (due to ALS progression))
|
19
|
|
Overall Study
Withdrawal Consent (due to other)
|
2
|
|
Overall Study
Death
|
28
|
|
Overall Study
Other
|
2
|
Baseline Characteristics
Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)
Baseline characteristics by cohort
| Measure |
300 mg Per Day
n=74 Participants
KNS-760704 (dexpramipexole) - 150 mg BID
|
|---|---|
|
Age, Continuous
|
57.4 years
STANDARD_DEVIATION 11.14 • n=93 Participants
|
|
Age, Customized
<=50
|
24 Participants
n=93 Participants
|
|
Age, Customized
51-65
|
30 Participants
n=93 Participants
|
|
Age, Customized
>65
|
20 Participants
n=93 Participants
|
|
Sex: Female, Male
Female
|
22 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
52 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
6 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
68 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
72 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Region of Enrollment
United States
|
74 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: 180 weeksPopulation: Number of subjects evaluated is the number of subjects who had a baseline assessment and at least one post-baseline assessment.
Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=74 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Number of Participants With Potentially Clinically Significant Hematology Results
WBC (<3.0 x 10^9/L)
|
5 Participants
|
|
Number of Participants With Potentially Clinically Significant Hematology Results
WBC (>=16.0 x 10^9/L)
|
2 Participants
|
|
Number of Participants With Potentially Clinically Significant Hematology Results
Neutrophils (<1.5 x 10^9/L)
|
3 Participants
|
|
Number of Participants With Potentially Clinically Significant Hematology Results
Neutrophils (>13.5 x 10^9/L)
|
4 Participants
|
PRIMARY outcome
Timeframe: 180 weeksPopulation: Number of subjects evaluated is the number of subjects who had a baseline assessment and at least one post-baseline assessment.
Number of participants with potentially clinically significant liver enzyme abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=74 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities
Alkaline phosphatase >2.5 times ULN (upper limit of the 'normal' reference range)
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities
ALT >3 times ULN
|
5 Participants
|
|
Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities
AST >3 times ULN
|
2 Participants
|
|
Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities
Total Bilirubin >1.5 times ULN
|
3 Participants
|
|
Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities
Creatinine >1.5 times ULN
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities
ALT or AST >3 times ULN and total bilirubin >2 times ULN (Hy's Law)
|
0 Participants
|
PRIMARY outcome
Timeframe: 180 weeksPopulation: Number of subjects evaluated is the number of subjects who had a baseline assessment and at least one post-baseline assessment.
Number of participants with potentially clinically significant ECG abnormalities for the safety population are presented. Percentages are based on the number of patients in the safety population who had at least one non-missing, post-baseline value.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=74 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Axis - LEFT AXIS DEVIATION
|
5 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Axis - RIGHT AXIS DEVIATION
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Conduction - LEFT ANTERIOR HEMIBLOCK
|
2 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Conduction - RSR' SUGGESTS RIGHT VENTRICULAR CONDUCTION DELAY
|
3 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Conduction - SHORT P-R INTERVAL
|
3 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Morphology - LEFT ATRIAL ENLARGEMENT
|
2 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Morphology - LEFT VENTRICULAR HYPERTROPHY BY VOLTAGE CRITERIA ONLY
|
2 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Morphology - LEFT VENTRICULAR HYPERTROPHY WITH REPOLARIZATION ABNORMALITIES
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Morphology - POSSIBLE RIGHT VENTRICULAR HYPERTROPHY
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Morphology - RIGHT VENTRICULAR HYPERTROPHY
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Myocardial Ischemia/Infarction - ANTEROSEPTAL MYOCARDIAL INFARCTION - AGE UNDETERMINED
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Myocardial Ischemia/Infarction - CANNOT RULE OUT POSSIBLE MYOCARDIAL INFARCTION (INFERIOR)
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Myocardial Isch/Infar. CANNOT RULE OUT POSSIBLE MYOCARDIAL INFARCTION MAY BE IMPROPER LEAD POSITIONS
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Myocardial Ischemia/Infarction - INFERIOR MYOCARDIAL INFARCTION - AGE UNDETERMINED
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Myocardial Ischemia/Infarction - OLD INFERIOR MYOCARDIAL INFARCTION
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Other - POOR PRECORDIAL R WAVE PROGRESSION. POSSIBLE IMPROPER PLACEMENT OF V LEADS.
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Other - POOR R WAVE PROGRESSION, PRECORDIAL LEADS
|
3 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
QT Interval - QTCB BORDERLINE PROLONGED
|
9 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Rhythm - ATRIAL FLUTTER WITH 3:1 VENTRICULAR RESPONSE
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Rhythm - FREQUENT VENTRICULAR PREMATURE BEATS
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Rhythm - RARE VENTRICULAR PREMATURE BEATS
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Rhythm - SINUS BRADYCARDIA
|
2 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Rhythm - SINUS TACHYCARDIA
|
25 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Rhythm - UNUSUAL P AXIS, POSSIBLE ECTOPIC ATRIAL RHYTHM
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
Rhythm - VENTRICULAR PREMATURE BEATS
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
ST-T Wave - COUNTERCLOCKWISE ROTATION
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
ST-T Wave - EARLY REPOLARIZATION
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
ST-T Wave - NON-SPECIFIC ST ABNORMALITY
|
2 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
ST-T Wave - NON-SPECIFIC ST AND T WAVE ABNORMALITY
|
1 Participants
|
|
Number of Participants With Potentially Clinically ECG Abnormalities
ST-T Wave - NON-SPECIFIC T WAVE ABNORMALITY
|
14 Participants
|
PRIMARY outcome
Timeframe: 180 weeksPopulation: Number of subjects evaluated is the number of subjects in the Safety Population who had a baseline assessment and at least one post-baseline assessment.
Number of participants with potentially clinically significant vital sign abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=74 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Temperature - >38 degree C. post-baseline and an increase from baseline of at least 1 degree Celsius
|
4 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Pulse Rate - >120 bpm post-baseline or an increase from baseline of >20 bpm
|
26 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Pulse Rate - >120 bpm post-baseline and an increase from baseline of >20 bpm
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Pulse Rate - <50 bpm post-baseline or a decrease from baseline of >20 bpm
|
9 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Pulse Rate - <50 bpm post-baseline and a decrease from baseline of >20 bpm
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Systolic Blood Pressure - >180 mmHg post-baseline or an increase from baseline of >40 mmHg
|
3 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Systolic Blood Pressure - >180 mmHg post-baseline and an increase from baseline of >40 mmHg
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Systolic Blood Pressure - <90 mmHg post-baseline or a decrease from baseline of >30 mmHg
|
11 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Systolic Blood Pressure - <90 mmHg post-baseline and a decrease from baseline of >30 mmHg
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Diastolic Blood Pressure - >105 mmHg post-baseline or an increase from baseline of >30 mmHg
|
4 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Diastolic Blood Pressure - >105 mmHg post-baseline and an increase from baseline of >30 mmHg
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Diastolic Blood Pressure - <50 mmHg post-baseline or a decrease from baseline of >20 mmHg
|
13 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Diastolic Blood Pressure - <50 mmHg post-baseline and a decrease from baseline of >20 mmHg
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Weight - Increase from baseline of >7%
|
4 Participants
|
|
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Weight - Decrease from baseline of >7%
|
32 Participants
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=69 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12
|
-2.48 units on a scale
Standard Deviation 3.424
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=55 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24
|
-4.29 units on a scale
Standard Deviation 4.589
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=45 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48
|
-7.49 units on a scale
Standard Deviation 8.476
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
Change in Percent Predicted Upright Vital Capacity from Baseline to Week 12. A negative change indicates clinical worsening.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=55 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Change in Upright Vital Capacity From Baseline to Week 12
|
-7.7 percentage of predicted vital capacity
Standard Deviation 14.03
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
Change in Percent Predicted Upright Vital Capacity from Baseline to Week 24. A negative change indicates clinical worsening.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=48 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Change in Upright Vital Capacity From Baseline to Week 24
|
-12.5 percentage of predicted vital capacity
Standard Deviation 18.90
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
Change in Percent Predicted Upright Vital Capacity from Baseline to Week 48. A negative change indicates clinical worsening.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=35 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Change in Upright Vital Capacity From Baseline to Week 48
|
-15.8 percentage of predicted vital capacity
Standard Deviation 17.59
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=69 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Change in McGill Single-Item Scale (SIS) From Baseline to Week 12
|
-0.1 units on a scale
Standard Deviation 1.48
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=54 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Change in McGill Single-Item Scale (SIS) From Baseline to Week 24
|
-0.5 units on a scale
Standard Deviation 2.05
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=45 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Change in McGill Single-Item Scale (SIS) From Baseline to Week 48
|
-0.8 units on a scale
Standard Deviation 1.76
|
SECONDARY outcome
Timeframe: 144 weeksPopulation: Randomized subjects who had at least 1 post-baseline clinical status evaluation. Subjects who had a feeding tube in place at the beginning of the study were not included in this analysis.
Number of participants who had a feeding tube placed during the study.
Outcome measures
| Measure |
KNS-760704 300 mg/Day
n=70 Participants
Open-label KNS-760704 (150 mg Q12H)
KNS-760704: 150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
|
|---|---|
|
Number of Subjects With Feeding Tube Placed During the Study.
|
24 participants
|
Adverse Events
300 mg Per Day
Serious events: 48 serious events
Other events: 74 other events
Deaths: 38 deaths
Serious adverse events
| Measure |
300 mg Per Day
n=74 participants at risk
KNS-760704 (dexpramipexole) - 150 mg BID
|
|---|---|
|
Blood and lymphatic system disorders
Neutropenia
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Cardiac disorders
Palpitations
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Colitis ischaemic
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Salivary hypersecretion
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Constipation
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Ileus
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Intestinal obstruction
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
General disorders
Non-cardiac chest pain
|
2.7%
2/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
General disorders
Oedema
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Hepatobiliary disorders
Cholecystitis acute
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Pneumonia
|
6.8%
5/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Pyelonephritis acute
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Tooth abscess
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Abscess limb
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Acinetobacter infection
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Bronchopneumonia
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Gastroenteritis
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Lung infection pseudomonal
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Pneumonia staphylococcal
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Tracheobronchitis
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Urosepsis
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Injury, poisoning and procedural complications
Fall
|
2.7%
2/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Injury, poisoning and procedural complications
Face injury
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Injury, poisoning and procedural complications
Facial bones fracture
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Injury, poisoning and procedural complications
Humerus fracture
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Injury, poisoning and procedural complications
Limb injury
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Metabolism and nutrition disorders
Failure to thrive
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Nervous system disorders
Amyotrophic lateral sclerosis
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Nervous system disorders
Syncope
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Psychiatric disorders
Delirium
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Psychiatric disorders
Delusion
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
33.8%
25/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory arrest
|
9.5%
7/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
4.1%
3/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Lung infiltration
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary infarction
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
2.7%
2/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
4.1%
3/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Upper airway obstruction
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Surgical and medical procedures
Wound drainage
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Vascular disorders
Deep vein thrombosis
|
1.4%
1/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
Other adverse events
| Measure |
300 mg Per Day
n=74 participants at risk
KNS-760704 (dexpramipexole) - 150 mg BID
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
6.8%
5/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Cardiac disorders
Tachycardia
|
8.1%
6/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Constipation
|
36.5%
27/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Salivary hypersecretion
|
23.0%
17/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Dysphagia
|
13.5%
10/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Diarrhoea
|
14.9%
11/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
9.5%
7/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Nausea
|
14.9%
11/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Abdominal distension
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Ileus
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Gastrointestinal disorders
Vomiting
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
General disorders
Oedema peripheral
|
23.0%
17/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
General disorders
Fatigue
|
14.9%
11/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
General disorders
Pyrexia
|
17.6%
13/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
General disorders
Non-cardiac chest pain
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
General disorders
Pain
|
9.5%
7/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Urinary tract infection
|
25.7%
19/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Bronchitis
|
6.8%
5/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Nasopharyngitis
|
6.8%
5/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Pneumonia
|
13.5%
10/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Oral candidiasis
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Sinusitis
|
9.5%
7/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Infections and infestations
Upper respiratory tract infection
|
8.1%
6/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Injury, poisoning and procedural complications
Fall
|
32.4%
24/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Injury, poisoning and procedural complications
Contusion
|
10.8%
8/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Investigations
Weight decreased
|
14.9%
11/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Investigations
Blood triglycerides increased
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Investigations
White blood cell count increased
|
6.8%
5/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
6.8%
5/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
28.4%
21/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
9.5%
7/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
9.5%
7/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
8.1%
6/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.8%
5/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Nervous system disorders
Dysarthria
|
17.6%
13/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Nervous system disorders
Amyotrophic lateral sclerosis
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Nervous system disorders
Headache
|
8.1%
6/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Psychiatric disorders
Anxiety
|
13.5%
10/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Psychiatric disorders
Insomnia
|
17.6%
13/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Psychiatric disorders
Depression
|
9.5%
7/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Renal and urinary disorders
Urinary retention
|
10.8%
8/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
35.1%
26/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
29.7%
22/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
16.2%
12/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
6.8%
5/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory arrest
|
9.5%
7/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
5.4%
4/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Skin and subcutaneous tissue disorders
Decubitus ulcer
|
13.5%
10/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
8.1%
6/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
|
Skin and subcutaneous tissue disorders
Rash
|
9.5%
7/74 • Adverse events were monitored and reported from the first dose through 30 days following the last dose.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place