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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03626012 | A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 in Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis | PHASE1 | COMPLETED | 106 | — | — | Sep 10, 2018 | Nov 17, 2021 | Jan 14, 2022 | 21 | United States, Canada +4 |
An AE is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death, places participant at immediate risk of death, requires initial or prolonged inpatient hospitalization, results in persistent or significant disability/incapacity, results in congenital anomaly, is a medically important event.
| Arm | Type | Description |
|---|---|---|
| Cohort 1: BIIB078 First Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days. |
| Cohort 2: BIIB078 Second Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days. |
| Cohort 3: BIIB078 Third Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days. |
| Cohort 4: BIIB078 Fourth Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by five maintenance doses on five later days. |
| Cohort 5: BIIB078 Fifth Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by five maintenance doses on five later days. |
| Cohort 6: BIIB078 Sixth Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by five maintenance doses on five later days. |
| Cohorts 1-6: Placebo | PLACEBO_COMPARATOR | Matching placebo will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days (Cohorts 1 through 3) and five maintenance doses on five later days (Cohorts 4 through 6). |
| Name | Type | Description |
|---|---|---|
| BIIB078 | DRUG | Administered as specified in the treatment arm. |
| Placebo | DRUG | Administered as specified in the treatment arm. |
Key Inclusion Criteria: * Ability of the participant to understand the purpose and risks of the study, to provide signed and dated informed consent, and to authorize the use of confidential health information in accordance with national and local participant privacy regulations; or, in the event of...