Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BIIB078 · 1 trial · 1 indication
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...
BIIB078 was studied for the treatment of amyotrophic lateral sclerosis, or ALS, specifically in adults with C9ORF72-associated ALS. This form of ALS is linked to a genetic change in the C9ORF72 gene. The program was investigated in a single Phase 1 clinical trial that has since been completed.
BIIB078 was developed by Biogen Inc., which trades on the Nasdaq under the ticker BIIB. Biogen sponsored the clinical program evaluating the drug in adults with C9ORF72-associated amyotrophic lateral sclerosis. The company is a biotechnology firm focused on neurological and neurodegenerative diseases.
BIIB078 was in Phase 1 clinical development. It is an investigational agent and is not an approved therapy. The single Phase 1 trial of BIIB078 has been completed, and no trials of the drug are currently listed as active. The study enrolled 106 participants.
BIIB078 was evaluated in the Phase 1 trial NCT03626012, titled a study to assess the safety, tolerability, and pharmacokinetics of BIIB078 in adults with C9ORF72-associated amyotrophic lateral sclerosis. The completed trial enrolled 106 adults, was randomized, double-blind, and placebo-controlled, and ran in the United States, Canada, Ireland, the Netherlands, Switzerland, and the United Kingdom.
The BIIB078 Phase 1 trial NCT03626012 enrolled adults 18 years and older of any sex with C9ORF72-associated amyotrophic lateral sclerosis. Participants were selected based on the C9ORF72 genetic alteration, and healthy volunteers were not included. The study was randomized, double-blind, and placebo-controlled, with 106 participants enrolled.