Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04414345 | HEALEY ALS Platform Trial - Regimen C CNM-Au8 | PHASE2 | COMPLETED | 161 | — | — | Jul 30, 2020 | Mar 7, 2023 | Jul 25, 2023 | 1 | United States |
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.
| Arm | Type | Description |
|---|---|---|
| CNM-Au8 | EXPERIMENTAL | Drug: CNM-Au8 Administration: Oral Dosage: 30 mg or 60 mg daily |
| Matching Placebo | PLACEBO_COMPARATOR | Administration: Oral Dosage: 2 bottles daily |
| Name | Type | Description |
|---|---|---|
| CNM-Au8 | DRUG | Drug: CNM-Au8 Administration: Oral Dosage: 30 mg or 60 mg daily |
| Matching Placebo | DRUG | Drug: Matching Placebo Administration: Oral Dosage: 2 bottles daily |
Inclusion Criteria: * No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683). Exclusion Criteria: * The following exclusion criterion is in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683). 1. Histo...