Phase 2
Completed N=102
Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS)
Source: ClinicalTrials.gov NCT00647296 ↗Enrolled (actual)
102
Serious AEs
12.4%
Results posted
Jul 2021
Primary outcomePrimary: Part 1: Number of Participants With Potentially Clinically Significant Hematology Results by Treatment Group — 0; 0; 0; 0 Participants
Summary
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part 1: Number of Participants With Potentially Clinically Significant Hematology Results by Treatment Group |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Part 1: Number of Participants With Potentially Clinically Significant Blood Chemistry Results by Treatment Group |
0; 0; 0; 1; 0; 0 | — |
| PRIMARY Part 1: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Part 1: Number of Participants With Potentially Clinically Significant Vital Sign Measurements by Treatment Group |
0; 2; 0; 0; 2; 0 | — |
| SECONDARY Part 1: Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to Week 12 by Treatment Group |
-1.278; -1.885; -1.165; -0.878 | 0.1385 |
| SECONDARY Part 1: Slope of Upright Vital Capacity From Baseline to Week 12 by Treatment Group |
-4.398; -4.003; -2.389; -3.947 | 0.7973 |
| SECONDARY Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant Hematology |
0; 0; 0; 0; 0 | — |
| SECONDARY Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant Blood Chemistry Results |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Part 2 Placebo Washout: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant Vital Sign Measurements |
0; 0; 2; 0; 1; 1 | — |
| SECONDARY Part 2 Placebo Washout: Absolute Change in ALSFRS-R Total Score |
-1.2 | — |
| SECONDARY Part 2 Placebo Washout: Absolute Change in Upright Vital Capacity (Percent Predicted) From Baseline to End of Placebo Washout (Week 4) |
-3.1 | — |
| SECONDARY Part 2 Double-Blind Treatment: Number of Participants With Potentially Clinically Significant Hematology Results by Treatment Group |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Part 2 Double-Blind Treatment: Number of Participants With Potentially Clinically Significant Blood Chemistry Results by Treatment Group |
0; 0; 0; 0; 1; 2 | — |
| SECONDARY Part 2 Double-Blind Treatment: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group |
2; 0; 0; 0; 0; 0 | — |
| SECONDARY Part 2 Double-Blind Treatment: Number of Participants With Potentially Clinically Significant Vital Sign Measurements by Treatment Group |
0; 0; 0; 1; 2; 1 | — |
| SECONDARY Part 2 Double-Blind Treatment: Slope of the ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to Week 28 by Treatment Group |
-1.284; -1.021 | 0.1772 |
| SECONDARY Part 2 Double-Blind Treatment: Slope of Percent Predicted Upright Vital Capacity From Baseline by Treatment Group |
-2.452; -3.067 | 0.4025 |
Eligibility Criteria
Inclusion Criteria
- Patients with diagnosis of familial or sporadic ALS, defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria
- Patients with ALS symptom onset 65% of predicted for age, height, and gender
Exclusion Criteria
- Patients in whom causes of neuromuscular weakness other than ALS have not been excluded
- Patients without clinical evidence of upper motor neuron dysfunction
- Patients with clinically suspected ALS according to the World Federation of Neurology El Escorial criteria
- Patients with prior exposure to KNS-760704 or the R(+) enantiomer of pramipexole (i.e., R(+)-pramipexole)
- Patients taking other investigational agents (including lithium) within 30 days of randomization or during the study
Data sourced from ClinicalTrials.gov (NCT00647296). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.