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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04856982 | A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation | PHASE3 | ACTIVE NOT_RECRUITING | 158 | — | — | May 17, 2021 | Apr 30, 2028 | Mar 30, 2026 | 32 | United States, Australia +12 |
| Arm | Type | Description |
|---|---|---|
| Part A: Natural History Run-in | NO_INTERVENTION | Participants enrolled in Part A will undergo blood draws approximately once every 28 days to assess neurofilament light chain (NfL) levels. |
| Part B: Randomized, Double-Blind, Placebo-Controlled | EXPERIMENTAL | Participants from Part A who meet the protocol-defined NfL threshold and remain presymptomatic may be eligible to participate in Part B. During Part B, participants will receive tofersen 100 milligram (mg) or placebo via intrathecal (IT) injection on Days 1, 15, 29, and every 28 days thereafter for up to approximately 5.6 years. |
| Part C: Open-Label Extension | EXPERIMENTAL | Participants from Part B who develop clinically manifest ALS may be eligible to participate in Part C. During Part C, participants who received placebo in Part B will receive tofersen 100 mg via IT injection on Days 1, 15, 29, and every 28 days thereafter up to the final maintenance dost visit. Participants who received tofersen during Part B will receive tofersen 100 mg on Days 1, 29, and every 28 days thereafter up to the final maintenance dost visit, with a dose of placebo on Day 15 to maintain the study blind. The combined duration of Part B and Part C is up to approximately 5.6 years. |
| Part D: Open-Label Treatment | EXPERIMENTAL | Participants from Part A who develop clinically manifest ALS prior to randomization in Part B may be eligible to participate in Part D. During Part D, participants will receive tofersen100 mg via IT injection on Days 1, 15, 29, and every 28 days thereafter for up to 2 years. |
| Name | Type | Description |
|---|---|---|
| Tofersen | DRUG | Administered as specified in the treatment arm |
| Placebo | DRUG | Administered as specified in the treatment arm |
Key Part A Inclusion Criteria: * Participants should have a protocol-defined rapidly progressive SOD1 mutation, confirmed by a central reader, or a SOD1 mutation that is approved for inclusion by an external mutation adjudication committee. * Participants with plasma NfL level less than the protoco...