Phase 3
Completed N=942
Phase 3 Study of Dexpramipexole in ALS
Source: ClinicalTrials.gov NCT01281189 ↗Enrolled (actual)
942
Serious AEs
48.6%
Results posted
Jun 2021
Primary outcomePrimary: Composite Assessment of Function and Survival (CAFS) at 12 Months — 438.84; 441.76 units on a scale — p=0.8568
◆ Published Evidence
Highly cited
235citations · ~18 / year
Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial.
Summary
The purpose of this study is to determine whether dexpramipexole (150 mg twice daily) is safe and effective in the treatment of Amyotrophic Lateral Sclerosis (ALS).
Linked Publications
-
Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Composite Assessment of Function and Survival (CAFS) at 12 Months |
438.84; 441.76 | 0.8568 |
| PRIMARY Death up to 12 Months (CAFs Individual Component) |
17.2; 16.0 | 0.8375 |
| PRIMARY Change From Baseline in ALSFRS-R at 12 Months (CAFs Individual Component) |
-13.415; -13.339 | 0.9019 |
| SECONDARY Death or Respiratory Insufficiency (DRI) up to Month 18 |
27.2; 22.3 | 0.7715 |
| SECONDARY Death up to 18 Months |
23.1; 20.5 | 0.9033 |
| SECONDARY ≤50% Predicted Upright Slow Vital Capacity (SVC) or Died up to 18 Months |
41.9; 36.5 | 0.7720 |
Eligibility Criteria
Inclusion Criteria
- Aged 18 to 80 years old, inclusive, on Day 1.
- Diagnosis of sporadic or familial ALS.
- Onset of first ALS symptoms within 24 months prior to Day 1.
- World Federation of Neurology El Escorial criteria are met for a possible, laboratory-supported probable, probable, or definite ALS diagnosis.
- Upright slow vital capacity (SVC) of 65% or more at screening.
- Patients taking or not taking Riluzole are eligible for this study: if a patient has never taken Riluzole, he or she is eligible; if a patient is currently taking Riluzole, he or she must have been on a stable dose for at least 60 days; if a patient has discontinued Riluzole, he or she must have stopped taking it for at least 30 days.
- Must be able to swallow tablets at the time of study entry.
Exclusion Criteria
- Other medically significant illness.
- Clinically significant abnormal laboratory values.
- Pregnant women or women breastfeeding.
- Prior exposure to dexpramipexole.
- Currently taking pramipexole or other dopamine agonists.
Other protocol-defined inclusion/exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT01281189) and the linked publication. 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.