Table 3.
Currently tested compounds and compounds used in studies that are recruiting participants.
| Compound | Endpoints § | Outcome of Previous Studies in ALS Patients | Reference |
|---|---|---|---|
| TUDCA | ALSFRS-R | success | [330] |
| Fasudil | survival, ALSFRS-R | - | [332] |
| Pimozide | ALSFRS-R, SVC | failure | [346] |
| Methylcobalamin | survival, ALSFRS-R | success | [336] |
| Deferiprone | CAFS | Mixed * | [337] |
| Cu(II)ATSM | ALSFRS-R, ECAS, SVC | success | - |
| Arimoclomol | CAFS | Failure ** | [341] |
| Ibudilast | ALSFRS-R, ALSAQ-5 | success | - |
| RNS60 | SVC, ALSFRS-R | failure | [345] |
§—primary endpoints, secondary endpoints in studies which primarily assessed safety and tolerability. *—study reported statistically significant differences in ALSFRS-R and body-mass index (BMI) decline between the three-month treatment-free period compared to the first three months of treatment. **—this study has shown a favorable trend in decreasing the decline of ALSFRS-R, FEV6 (Forced Expiratory Volume in six seconds), and CAFS, although this trend was not statistically significant.