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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03945279 | A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis | PHASE1 | COMPLETED | 49 | — | — | May 30, 2019 | Jun 21, 2021 | Apr 18, 2023 | 9 | United States |
An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, or is a medically important event.
| Arm | Type | Description |
|---|---|---|
| Cohort 1: BIIB100 Dose 1 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 2: BIIB100 Dose 2 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 3: BIIB100 Dose 3 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 4: BIIB100 Dose 4 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 5: BIIB100 Dose 5 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 6: BIIB100 Dose 6 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 1-6: Matching Placebo | PLACEBO_COMPARATOR | Participants will receive single oral dose of matching placebo on Day 1. |
| Name | Type | Description |
|---|---|---|
| BIIB100 | DRUG | Administered as specified in the treatment arm. |
| Placebo | DRUG | Administered as specified in the treatment arm. |
Key Inclusion Criteria: * Must meet the laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria. * Participants taking concomitant riluzole at study entry must be on a stable dose for greater than or equals...