Trial Outcomes & Findings for Phase 3 Extension Study of Dexpramipexole in ALS (NCT NCT01622088)

NCT ID: NCT01622088

Last Updated: 2022-05-03

Results Overview

The number of subjects who reported an adverse event during the study

• Recruitment status: TERMINATED
• Study phase: PHASE3
• Target enrollment: 616 participants
• Primary outcome timeframe: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
• Results posted on: 2022-05-03

Participant Flow

Participant milestones

Measure	Dexpramipexole 300 mg/Day Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Overall Study STARTED	616
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	616

Reasons for withdrawal

Measure	Dexpramipexole 300 mg/Day Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Overall Study Adverse Event	20
Overall Study Lost to Follow-up	1
Overall Study Withdrawal by Subject	20
Overall Study Investigator Decision	1
Overall Study Death	53
Overall Study STUDY TERMINATED EARLY BY SPONSOR	521

Baseline Characteristics

One subject did not have age at Baseline reported.

Baseline characteristics by cohort

Measure	Dexpramipexole 150 mg BID n=616 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Age, Continuous	56.5 years STANDARD_DEVIATION 11.48 • n=615 Participants • One subject did not have age at Baseline reported.
Age, Customized <50 years	164 Participants n=616 Participants
Age, Customized between 50-65 years	305 Participants n=616 Participants
Age, Customized >65 years	146 Participants n=616 Participants
Age, Customized missing	1 Participants n=616 Participants
Sex: Female, Male Female	206 Participants n=616 Participants
Sex: Female, Male Male	410 Participants n=616 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=616 Participants
Race (NIH/OMB) Asian	6 Participants n=616 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=616 Participants
Race (NIH/OMB) Black or African American	6 Participants n=616 Participants
Race (NIH/OMB) White	585 Participants n=616 Participants
Race (NIH/OMB) More than one race	0 Participants n=616 Participants
Race (NIH/OMB) Unknown or Not Reported	19 Participants n=616 Participants
Region of Enrollment Canada	39 participants n=616 Participants
Region of Enrollment Netherlands	26 participants n=616 Participants
Region of Enrollment Sweden	13 participants n=616 Participants
Region of Enrollment Belgium	21 participants n=616 Participants
Region of Enrollment United States	279 participants n=616 Participants
Region of Enrollment Ireland	14 participants n=616 Participants
Region of Enrollment United Kingdom	45 participants n=616 Participants
Region of Enrollment Australia	26 participants n=616 Participants
Region of Enrollment France	33 participants n=616 Participants
Region of Enrollment Germany	76 participants n=616 Participants
Region of Enrollment Spain	44 participants n=616 Participants
Weight (kg)	73.94 kg STANDARD_DEVIATION 16.528 • n=154 Participants • Weight was unable to be measured for many subjects due to ALS disease impairment (e.g., use of a mobility device).
Height (cm)	173.1 cm STANDARD_DEVIATION 9.59 • n=616 Participants
Body mass index (kg/m^2)	24.65 kg/m^2 STANDARD_DEVIATION 5.058 • n=154 Participants • Weight, and therefore BMI, was unable to be measured for many subjects due to ALS disease impairment (e.g., use of a mobility device).

PRIMARY outcome

Timeframe: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

Population: The safety population is defined as all subjects who received at least 1 dose of study treatment

The number of subjects who reported an adverse event during the study

Outcome measures

Measure	Dexpramipexole 150 mg BID n=616 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Number of Subjects Who Reported an Adverse Event	454 Participants

PRIMARY outcome

Timeframe: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

Population: The safety population is defined as all subjects who received at least 1 dose of study treatment

The number of subjects enrolled who reported a serious adverse event during the study

Outcome measures

Measure	Dexpramipexole 150 mg BID n=616 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Number of Subjects Who Experienced a Serious Adverse Event	152 Participants

PRIMARY outcome

Timeframe: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

Population: The safety population is defined as all subjects who received at least 1 dose of study treatment

The number of subjects enrolled who discontinued the study treatment due to an adverse event during the study

Outcome measures

Measure	Dexpramipexole 150 mg BID n=616 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event	30 Participants

PRIMARY outcome

Timeframe: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

Population: The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline vital sign measurement during the study.

Number of Participants with Potentially Clinically Significant Vital Sign Abnormalities. 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	Dexpramipexole 150 mg BID n=500 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Number of Participants With Potentially Clinically Significant Vital Sign Results Systolic BP >180 mmHg	2 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Systolic BP Increase from Baseline >40 mmHg	6 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Systolic BP <90 mmHg	4 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Systolic BP Decrease from Baseline >30 mmHg	10 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Diastolic BP >105 mmHg	20 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Diastolic BP increase from Baseline >30 mmHg	5 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Diastolic BP <50 mmHg	2 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Diastolic BP Decrease from Baseline >20 mmHg	21 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Pulse >120 bpm	3 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Pulse Increase from Baseline >30 bpm	16 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Pulse <50 bpm	2 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Pulse Decrease from Baseline >20 bpm	30 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Temperature >38.5 C and Increase from Baseline>=1°C	1 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Weight >=7% Increase from Baseline	19 Participants
Number of Participants With Potentially Clinically Significant Vital Sign Results Weight >=7% Decrease from Baseline	37 Participants

PRIMARY outcome

Timeframe: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

Population: The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline hematology assessment during the study.

Number of Participants with Potentially Clinically Significant Hematology Results. 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	Dexpramipexole 150 mg BID n=590 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Number of Participants With Potentially Clinically Significant Hematology Results White Blood Cell Count < 3.0 x10^9 cells/L	18 Participants
Number of Participants With Potentially Clinically Significant Hematology Results White Blood Cell Count >= 16 x10^9 cells/L	6 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Total Absolute Neutrophil Count < 1.5 x10^9 cells/L	14 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Total Absolute Neutrophil Count >= 13.5 x10^9 cells/L	6 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Lymphocyte < 0.8 x10^9 cells/L	65 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Lymphocyte > 12 x10^9 cells/L	0 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Monocytes > 2.5 x10^9 cells/L	0 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Eosinophils > 1.6 x10^9 cells/L	0 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Basophils > 1.6 x10^9 cells/L	0 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Hemoglobin - Females <= 95 g/L	1 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Hemoglobin - Females >= 175 g/L	0 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Hemoglobin - Males <= 115 g/L	13 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Hemoglobin - Males >= 190 g/L	1 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Hematocrit - Females <= 32%	8 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Hematocrit - Females >= 54%	0 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Hematocrit - Males <= 37%	47 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Hematocrit - Males >= 60%	1 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Red Blood Cell Count <= 3.5 x10^12 cells/L	15 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Red Blood Cell Count >= 6.4 x10^12 cells/L	3 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Platelet Count <= 75 x10^9 cells/L	0 Participants
Number of Participants With Potentially Clinically Significant Hematology Results Platelet Count >= 700 x10^9 cells/L	3 Participants

PRIMARY outcome

Timeframe: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

Population: The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline blood chemistry assessment during the study.

Number of Participants with Potentially Clinically Significant Blood Chemistry Results. 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	Dexpramipexole 150 mg BID n=589 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Sodium <= 126 mmol/L	5 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Sodium >= 156 mmol/L	0 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Potassium <= 3 mmol/L	1 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Potassium >= 6 mmol/L	4 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Chloride <= 90 mmol/L	9 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Chloride >= 118 mmol/L	0 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Bicarbonate <= 16 mmol/L	7 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Bicarbonate >= 35 mmol/L	6 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Calcium <= 2 mmol/L	7 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Calcium >= 3 mmol/L	0 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Magnesium <= 0.5 mmol/L	0 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Magnesium >= 1.2 mmol/L	0 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Phosphorus <= 0.6 mmol/L	0 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Phosphorus >= 1.7 mmol/L	9 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Aspartate Aminotransferase - SGOT >= 3 x ULN U/L	4 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Alanine Aminotransferase - SGPT >= 3 x ULN U/L	12 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Alkaline Phosphatase >= 1.5 x ULN U/L	4 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Creatinine - Females >= 176.8 umol/L	1 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Creatinine - Males >= 176.8 umol/L	0 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Blood Urea Nitrogen >= 10.7 mmol/L	23 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Total Bilirubin >= 1.5 x ULN umol/L	8 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Total Protein <= 45 g/L	1 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Total Protein >= 100 g/L	0 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Albumin <= 25 g/L	3 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Uric Acid - Females >= 506 umol/L	1 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Uric Acid - Males >= 625 umol/L	4 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Glucose <= 2.2 mmol/L	0 Participants
Number of Participants With Potentially Clinically Significant Blood Chemistry Results Glucose >= 9.7 mmol/L	43 Participants

PRIMARY outcome

Timeframe: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

Population: The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline ECG assessment during the study.

Number of Participants with Potentially Clinically Significant ECG Results. 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	Dexpramipexole 150 mg BID n=460 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Number of Participants With Potentially Clinically Significant ECG Results QTcF > 450 ms	2 Participants
Number of Participants With Potentially Clinically Significant ECG Results QTcF > 480 ms	0 Participants
Number of Participants With Potentially Clinically Significant ECG Results QTcF > 500 ms	0 Participants
Number of Participants With Potentially Clinically Significant ECG Results QTcF Change from Baseline > 30 ms	34 Participants
Number of Participants With Potentially Clinically Significant ECG Results QTcF Change from Baseline > 60 ms	3 Participants
Number of Participants With Potentially Clinically Significant ECG Results QT > 450 ms	0 Participants
Number of Participants With Potentially Clinically Significant ECG Results QT > 480 ms	0 Participants
Number of Participants With Potentially Clinically Significant ECG Results QT > 500 ms	0 Participants
Number of Participants With Potentially Clinically Significant ECG Results QT Change from Baseline > 30 ms	59 Participants
Number of Participants With Potentially Clinically Significant ECG Results QT Change from Baseline > 60 ms	2 Participants
Number of Participants With Potentially Clinically Significant ECG Results QTcB > 480 ms	9 Participants
Number of Participants With Potentially Clinically Significant ECG Results QTcB > 450 ms	2 Participants
Number of Participants With Potentially Clinically Significant ECG Results QTcB > 500 ms	0 Participants
Number of Participants With Potentially Clinically Significant ECG Results QTcB Change from Baseline > 30 ms	69 Participants
Number of Participants With Potentially Clinically Significant ECG Results QTcB Change from Baseline > 60 ms	6 Participants
Number of Participants With Potentially Clinically Significant ECG Results PR > 220 ms and Percent Increase from Baseline >25%	0 Participants
Number of Participants With Potentially Clinically Significant ECG Results QRS > 110 ms and Percent Increase from Baseline >25%	1 Participants

SECONDARY outcome

Timeframe: Up to maximum 226 days: approximately 32.2 weeks

Population: Randomized subjects who had at least 1 post-baseline clinical evaluation

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 between 0 to 48, with higher scores representing better function. Slope is calculated using a linear mixed effects model with x-axis time in months and y-axis ALSFRS-R score. Units for slope are change per month in units on the ALSFRS-R scale.

Outcome measures

Measure	Dexpramipexole 150 mg BID n=607 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study	-0.7355 Change per month in ALSFRS-R Standard Error 0.04448

SECONDARY outcome

Timeframe: Up to maximum 226 days: approximately 32.2 weeks

Population: Randomized subjects who had at least 1 post-baseline clinical evaluation

SNIP is a test of inspiratory force (sternocleidomastoid and diaphragm) measured via a nasal cannula and is used to assess respiratory muscle weakness and to monitor changes in respiratory muscle strength over time. During the SNIP maneuver, the patient is asked to perform a strong, sharp, maximal sniff, whereby nasal pressure is measured via nasal cannula. The maximum recorded value after several attempts, with rest in between attempts, was use in the analysis.

Outcome measures

Measure	Dexpramipexole 150 mg BID n=610 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study	-0.1895 Change per month in SNIP (in cm H20) Standard Error 0.09933

SECONDARY outcome

Timeframe: 6 Months

Population: Subjects who were randomized, received at least 1 dose of study treatment, had at least 1 available post-dosing efficacy evaluation (ALSFRS-R) or died during the study period.

Kaplan-Meier estimate of percentage of subjects who died up to 6 months

Outcome measures

Measure	Dexpramipexole 150 mg BID n=615 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Death up to 6 Months	13.95 percentage of participants Interval 10.83 to 17.07

SECONDARY outcome

Timeframe: 6 months

Population: Subjects who were randomized, received at least 1 dose of study treatment, had at least 1 available post-dosing efficacy evaluation (ALSFRS-R) or died during the study period.

Kaplan-Meier estimate of percentage of subjects who died or had a death equivalent event (tracheostomy or permanent assisted ventilation [PAV], defined as use of noninvasive ventilation [NIV] for ≥22 hours per day for ≥10 days) up to 6 months

Outcome measures

Measure	Dexpramipexole 150 mg BID n=615 Participants Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Percentage of Participants With Death or Death Equivalent up to 6 Months	16.35 percentage of participants Interval 13.0 to 19.7

Adverse Events

Dexpramipexole 300 mg/Day

Serious events: 152 serious events

Other events: 215 other events

Deaths: 73 deaths

Serious adverse events

Measure	Dexpramipexole 300 mg/Day n=616 participants at risk Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Blood and lymphatic system disorders FEBRILE NEUTROPENIA	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Blood and lymphatic system disorders LEUKOPENIA	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Blood and lymphatic system disorders NEUTROPENIA	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Cardiac disorders CARDIAC ARREST	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Cardiac disorders CARDIO-RESPIRATORY ARREST	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Cardiac disorders STRESS CARDIOMYOPATHY	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Cardiac disorders VENTRICULAR FIBRILLATION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Gastrointestinal disorders COLITIS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Gastrointestinal disorders CONSTIPATION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Gastrointestinal disorders DYSPHAGIA	2.6% 16/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Gastrointestinal disorders FAECALOMA	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Gastrointestinal disorders GASTROINTESTINAL HAEMORRHAGE	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
General disorders DEATH	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
General disorders EUTHANASIA	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
General disorders GENERAL PHYSICAL HEALTH DETERIORATION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
General disorders HAEMORRHOIDS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
General disorders PYREXIA	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Hepatobiliary disorders CHOLECYSTITIS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations BRONCHITIS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations CATHETER SITE INFECTION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations CLOSTRIDIUM DIFFICILE COLITIS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations ENTEROCOCCAL INFECTION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations ENTEROCOCCAL SEPSIS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations ENTEROCOLITIS INFECTIOUS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations GASTROENTERITIS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations HERPES ZOSTER OTICUS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations LOWER RESPIRATORY TRACT INFECTION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations ORAL INFECTION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations PNEUMONIA	2.4% 15/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations PNEUMONIA PNEUMOCOCCAL	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations PSEUDOMONAL BACTERAEMIA	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations RESPIRATORY TRACT INFECTION	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Infections and infestations UROSEPSIS	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Injury, poisoning and procedural complications CAPNOTHORAX	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Injury, poisoning and procedural complications CERVICAL VERTEBRAL FRACTURE	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Injury, poisoning and procedural complications FALL	0.49% 3/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Injury, poisoning and procedural complications FEMUR FRACTURE	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Injury, poisoning and procedural complications HIP FRACTURE	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Investigations BLOOD GLUCOSE INCREASED	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Investigations LIVER FUNCTION TEST ABNORMAL	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Investigations SLEEP STUDY	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Investigations WEIGHT DECREASED	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Metabolism and nutrition disorders HYPOKALAEMIA	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Metabolism and nutrition disorders HYPONATRAEMIA	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Musculoskeletal and connective tissue disorders JOINT SWELLING	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) BRONCHIAL CARCINOMA	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Nervous system disorders AMYOTROPHIC LATERAL SCLEROSIS	2.6% 16/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Psychiatric disorders ANXIETY	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Psychiatric disorders CONFUSIONAL STATE	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Psychiatric disorders HALLUCINATION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Renal and urinary disorders CALCULUS URETERIC	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Renal and urinary disorders HYDRONEPHROSIS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Renal and urinary disorders URINARY RETENTION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders ACUTE RESPIRATORY FAILURE	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders ASPIRATION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders CHRONIC RESPIRATORY FAILURE	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders COUGH	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders DYSPNOEA	0.65% 4/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders FOREIGN BODY ASPIRATION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders HYPOVENTILATION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders LARYNGOSPASM	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders NASAL SEPTUM DEVIATION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders PNEUMONIA ASPIRATION	0.81% 5/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders PNEUMOTHORAX	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders PULMONARY EMBOLISM	0.49% 3/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders RESPIRATORY ARREST	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders RESPIRATORY DISORDER	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders RESPIRATORY DISTRESS	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders RESPIRATORY FAILURE	8.1% 50/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders RESPIRATORY MUSCLE WEAKNESS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Skin and subcutaneous tissue disorders ANGIOEDEMA	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Skin and subcutaneous tissue disorders RASH GENERALISED	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Social circumstances RESPITE CARE	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Surgical and medical procedures GASTROSTOMY TUBE INSERTION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Surgical and medical procedures INTESTINAL RESECTION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Surgical and medical procedures MECHANICAL VENTILATION	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Surgical and medical procedures TRACHEOSTOMY	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Vascular disorders DEEP VEIN THROMBOSIS	0.32% 2/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Vascular disorders SHOCK	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Vascular disorders THROMBOSIS	0.16% 1/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.

Other adverse events

Measure	Dexpramipexole 300 mg/Day n=616 participants at risk Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months
Infections and infestations URINARY TRACT INFECTION	5.0% 31/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Respiratory, thoracic and mediastinal disorders RESPIRATORY FAILURE	8.4% 52/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Gastrointestinal disorders CONSTIPATION	8.0% 49/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Gastrointestinal disorders NAUSEA	5.4% 33/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
General disorders OEDEMA PERIPHERAL	5.0% 31/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Injury, poisoning and procedural complications FALL	11.0% 68/616 • Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.

Additional Information

Head of Regulatory

Knopp Biosciences

Phone: 4124881776

Email: greg@knoppbio.com

Results disclosure agreements

• Principal investigator is a sponsor employee Sponsor must be consulted
• Publication restrictions are in place

Restriction type: OTHER