Phase 2
Completed N=90
Open Label Extension Study of AMX0035 in Patients With ALS
Source: ClinicalTrials.gov NCT03488524 ↗Enrolled (actual)
90
Serious AEs
34.4%
Results posted
Aug 2025
Primary outcomePrimary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 81 Participants
Summary
This extension study, in which all participants received active treatment (AMX0035), was designed to assess the longer-term safety and therapeutic potential of AMX0035 for participants who have completed the Main Study (AMX3500, also known as CENTAUR).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs) |
81 | — |
| SECONDARY Amyotrophic Lateral Sclerosis Rating Scale Revised Total Score (ALSFRS-R) Change in Slope |
17.38; 21.61 | 0.0239 sig |
| SECONDARY Survival - Time to Death |
18.7; 23.5 | 0.0475 sig |
| SECONDARY Composite of Time to Hospitalization, Death or Death Equivalent |
10.0; 14.8 | 0.0308 sig |
| SECONDARY Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Upper Extremities |
12.06; 19.83 | 0.0291 sig |
| SECONDARY Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Lower Extremities |
20.48; 25.24 | 0.2261 |
| SECONDARY Slow Vital Capacity Change in Slope |
37.85; 48.52 | 0.0372 sig |
| SECONDARY Accurate Testing of Limb Isometric Strength (ATLIS) Total Score Change in Slope |
16.65; 22.84 | 0.0503 |
Eligibility Criteria
Inclusion Criteria
- Completion of all visits in the randomized, double blind AMX3500 study. Participants who received tracheostomy or permanent assisted ventilation (PAV) during the course of the main study could enroll in the OLE if they completed all visits in the main study.
- Must enroll in the OLE within 28 days of the Week 24 visit of the main study.
- Signed informed consent to enter the OLE phase.
Exclusion Criteria
- Discontinued study drug prematurely in the double-blind phase of the study for reasons other than tracheostomy or PAV.
- Exposure to or anticipated requirement for any disallowed medication listed in the protocol.
- Any ongoing adverse events that in the opinion of the Site Investigator are clear contraindications to the study drug.
- Unstable cardiac or other life-threatening disease emergent during the randomized, double blind study
- Any major medical condition that in the opinion of the Site Investigator would interfere with the study and place the subject at increased risk.
Data sourced from ClinicalTrials.gov (NCT03488524). 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.