Phase 3
Completed N=239
Arimoclomol in Amyotropic Lateral Sclerosis
Source: ClinicalTrials.gov NCT03491462 ↗Enrolled (actual)
239
Serious AEs
23.4%
Results posted
Aug 2023
Primary outcomePrimary: Combined Assessment of Function and Survival (CAFS) — 0.51; 0.49 score on a scale — p=0.6208
◆ Published Evidence
Established
32citations · ~16 / year
Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial.
Summary
A multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS)
Linked Publications
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Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Combined Assessment of Function and Survival (CAFS) |
0.51; 0.49 | 0.6208 |
| SECONDARY Time to Permanent Assisted Ventilation (PAV) / Tracheostomy / Death |
NA; NA | — |
| SECONDARY Change From Baseline to Week 76 (or End-of-trial) in the Revised ALS Functional Rating Scale (ALSFRS-R) |
-15.4; -15.0 | — |
| SECONDARY Change From Baseline to Week 76 (or End-of-trial) in Percent (%) Predicted Slow Vital Capacity (SVC) |
-30.65; -30.38 | — |
Eligibility Criteria
Inclusion Criteria
- Subject meets revised El Escorial criteria for clinically possible, clinically probable / clinically probable ALS laboratory-supported, clinically definite ALS or clinically definite familial ALS laboratory-supported
- 18 months or less since first appearance of weakness (e.g. limb weakness, dysarthria, dysphagia, shortness of breath)
- ALS Functional Rating Scale-Revised (ALSFRS-R) equal to or above 35 and erect (seated) Slow Vital Capacity (SVC) % predicted equal to or above 70% at Screening
Exclusion Criteria
- Tracheostomy or use of non-invasive ventilation for more than 2 hours during waking hours at the time of Screening and Baseline
- Pregnant or breast-feeding
- Current or anticipated use of diaphragmatic pacing
- Any other relevant medically significant condition which could present risk to the subject or interfere with the assessment of safety or has an increased risk of causing death during the trial
Data sourced from ClinicalTrials.gov (NCT03491462) 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.