Adults 21 to 80, any sex, with Amyotrophic Lateral Sclerosis. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Part 1: Number of Participants With Potentially Clinically Significant Hematology Results by Treatment GroupPrimary· 12 weeks
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.
White Blood Cell Count (x10^3/uL) < 2000/uL
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Neutrophils (x10^3/uL) < 1000/uL
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Eosinophils (x10^3/uL) > 5000/uL
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Hemoglobin (g/dL) < 8 g/dL
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Platelets (x10^3/uL) < 50,000/uL
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Part 1: Number of Participants With Potentially Clinically Significant Blood Chemistry Results by Treatment GroupPrimary· 12 weeks
Number of Participants with Potentially Clinically Significant Blood Chemistry 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.
Creatine Kinase (U/L) >= 10xULN
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
1
Total Bilirubin (mg/dL) > 1.5xULN
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
ALT (SGPT) (U/L) > 3xULN
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
2
AST (SGOT) (U/L) > 3xULN
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
1
Alkaline Phosphatase (U/L) > 1.5xULN
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
1
BUN (mg/dL) > 5xULN
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Creatinine (mg/dL) > 3xULN
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Sodium (mEq/L) low < 123 mEq/L
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Part 1: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment GroupPrimary· 12 weeks
Number of Participants with Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group. Percentages are based on the number of patients with at least one non-missing post-baseline value in each treatment group.
Rhythm Sinus Tachycardia > 120 bpm
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Rhythm Sinus Bradycardia < 40 bpm
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Rhythm Sinus Block/Sinus Arrest
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Rhythm Junctional Rhythm
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Rhythm Atrial Flutter
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Rhythm Atrial Fibrillation
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
1
Rhythm Supraventricular
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Rhythm Tachycardia > 100 bpm
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Part 1: Number of Participants With Potentially Clinically Significant Vital Sign Measurements by Treatment GroupPrimary· 12 weeks
Number of Participants with Potentially Clinically Significant Vital Sign Measurements by Treatment Group. Percentages are based on the number of patients with at least one non-missing post-baseline value in each treatment group.
Systolic Blood Pressure Increase of > 20 mmHg from baseline and >= 180mmHg
Group
Value
95% CI
Placebo
0
50 mg/Day
2
150 mg/Day
0
300 mg/Day
0
Systolic Blood Pressure Decrease of < -20 mmHg from baseline and <= 90 mmHg
Group
Value
95% CI
Placebo
2
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Diastolic Blood Pressure Increase of > 15 mmHg from baseline and >= 105 mmHg
Group
Value
95% CI
Placebo
0
50 mg/Day
1
150 mg/Day
0
300 mg/Day
0
Diastolic Blood Pressure Decrease of < -15 mmHg from baseline and <= 50 mmHg
Group
Value
95% CI
Placebo
1
50 mg/Day
1
150 mg/Day
0
300 mg/Day
0
Pulse Increase of > 15 bpm from baseline and >= 120
Group
Value
95% CI
Placebo
0
50 mg/Day
1
150 mg/Day
0
300 mg/Day
0
Pulse Decrease of < -15 bpm from baseline and <= 50
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
1
Body Weight Increase of > 7% lbs from baseline
Group
Value
95% CI
Placebo
0
50 mg/Day
0
150 mg/Day
0
300 mg/Day
0
Body Weight Decrease of < -7% lbs from baseline
Group
Value
95% CI
Placebo
3
50 mg/Day
2
150 mg/Day
0
300 mg/Day
1
Part 1: Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to Week 12 by Treatment GroupSecondary· 12 weeks
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, 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.
Group
Value
95% CI
Placebo
-1.278
-1.82 – -0.74
Dexpramipexole (50 mg/Day)
-1.885
-2.48 – -1.29
Dexpramipexole (150 mg/Day)
-1.165
-1.71 – -0.62
Dexpramipexole (300 mg/Day)
-0.878
-1.44 – -0.31
Part 1: Slope of Upright Vital Capacity From Baseline to Week 12 by Treatment GroupSecondary· 12 weeks
Slope of change in Upright Vital Capacity (percent predicted upright vital capacity) from Baseline to Week 12. A negative change/slope indicates clinical worsening. Slope is calculated using a linear mixed effects model with x-axis time in months and y-axis as percent predicted upright vital capacity. Units for slope are change per month in percent predicted upright vital capacity.
Group
Value
95% CI
Placebo
-4.398
-6.42 – -2.38
Dexpramipexole (50 mg/Day)
-4.003
-6.23 – -1.77
Dexpramipexole (150 mg/Day)
-2.389
-4.44 – -0.34
Dexpramipexole (300 mg/Day)
-3.947
-6.07 – -1.82
Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant HematologySecondary· 4 weeks
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.
White Blood Cell Count (x10^3/uL) < 2000/uL
Group
Value
95% CI
Placebo
0
Neutrophils (x10^3/uL) < 1000/uL
Group
Value
95% CI
Placebo
0
Eosinophils (x10^3/uL) > 5000/uL
Group
Value
95% CI
Placebo
0
Hemoglobin (g/dL) < 8 g/dL
Group
Value
95% CI
Placebo
0
Platelets (x10^3/uL) < 50,000/uL
Group
Value
95% CI
Placebo
0
Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant Blood Chemistry ResultsSecondary· 4 weeks
Number of Participants with Potentially Clinically Significant Blood Chemistry 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.
Creatine Kinase (U/L) >= 10xULN
Group
Value
95% CI
Placebo
0
Total Bilirubin (mg/dL) > 1.5xULN
Group
Value
95% CI
Placebo
0
ALT (SGPT) (U/L) > 3xULN
Group
Value
95% CI
Placebo
0
AST (SGOT) (U/L) > 3xULN
Group
Value
95% CI
Placebo
0
Alkaline Phosphatase (U/L) > 1.5xULN
Group
Value
95% CI
Placebo
0
BUN (mg/dL) > 5xULN
Group
Value
95% CI
Placebo
0
Creatinine (mg/dL) > 3xULN
Group
Value
95% CI
Placebo
0
Sodium (mEq/L) low < 123 mEq/L
Group
Value
95% CI
Placebo
0
Part 2 Placebo Washout: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) FindingsSecondary· 4 weeks
Number of Participants with Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group. Percentages are based on the number of patients with at least one non-missing post-baseline value in each treatment group.
Rhythm Sinus Tachycardia > 120 bpm
Group
Value
95% CI
Placebo
0
Rhythm Sinus Bradycardia < 40 bpm
Group
Value
95% CI
Placebo
0
Rhythm Sinus Block/Sinus Arrest
Group
Value
95% CI
Placebo
0
Rhythm Junctional Rhythm
Group
Value
95% CI
Placebo
0
Rhythm Atrial Flutter
Group
Value
95% CI
Placebo
0
Rhythm Atrial Fibrillation
Group
Value
95% CI
Placebo
0
Rhythm Supraventricular
Group
Value
95% CI
Placebo
0
Rhythm Tachycardia > 100 bpm
Group
Value
95% CI
Placebo
0
Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant Vital Sign MeasurementsSecondary· 4 weeks
Number of Participants with Potentially Clinically Significant Vital Sign Measurements by Treatment Group. Percentages are based on the number of patients with at least one non-missing post-baseline value in each treatment group.
Systolic Blood Pressure Increase of > 20 mmHg from baseline and >= 180mmHg
Group
Value
95% CI
Placebo
0
Systolic Blood Pressure Decrease of < -20 mmHg from baseline and <= 90 mmHg
Group
Value
95% CI
Placebo
0
Diastolic Blood Pressure Increase of > 15 mmHg from baseline and >= 105 mmHg
Group
Value
95% CI
Placebo
2
Diastolic Blood Pressure Decrease of < -15 mmHg from baseline and <= 50 mmHg
Group
Value
95% CI
Placebo
0
Pulse Increase of > 15 bpm from baseline and >= 120
Group
Value
95% CI
Placebo
1
Pulse Decrease of < -15 bpm from baseline and <= 50
Group
Value
95% CI
Placebo
1
Body Weight Increase of > 7% lbs from baseline
Group
Value
95% CI
Placebo
0
Body Weight Decrease of < -7% lbs from baseline
Group
Value
95% CI
Placebo
0
Part 2 Placebo Washout: Absolute Change in ALSFRS-R Total ScoreSecondary· 4 weeks
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, with higher scores representing better function.
Units are points on the ALSFRS-R score as an absolute change from the baseline of the placebo washout to week 4 of the placebo washout.
Group
Value
95% CI
Placebo
-1.2
± 0.27
Part 2 Placebo Washout: Absolute Change in Upright Vital Capacity (Percent Predicted) From Baseline to End of Placebo Washout (Week 4)Secondary· 4 weeks
Absolute change in Upright Vital Capacity From Baseline to Week 4. Units are percent of predicted Upright Vital Capacity. A negative change indicates clinical worsening.
Group
Value
95% CI
Placebo
-3.1
± 0.95
Adverse events — posted to ClinicalTrials.gov
Time frame: Part 1: up to 12 weeks; Part 2 Placebo washout: up to 4 weeks; Part 2 Treatment period: up to 18 months.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This was a 2-part study of dexpramipexole in patients with ALS.
Part 1 was a randomized, placebo-controlled, multi-center study to evaluate the safety, tolerability, and clinical effects of oral administration of 3 dosage levels of dexpramipexole vs. placebo for 12 weeks.
Part 2 was a randomized, double-blind, 2-arm, parallel group, extension study evaluating the safety, tolerability, and clinical effects of oral administration of 2 dosage levels of dexpramipexole for up to 72 weeks.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Knopp Biosciences
Last refreshed: 8 July 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00647296.