A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

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NCT04856982 · readout in 352 d

Sponsored by Biogen (industry) · BIIB — their whole pipeline →.

Phase

Phase 3

Status

Active, not recruiting

Study type

Interventional

Design

Randomized · Triple · Treatment

Enrollment

158actual

Sites

Countries

Australia, Belgium, Brazil +11

Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →

Source: ClinicalTrials.gov, retrieved 2026-08-19

Dates

each axis at the precision it was filed, with the registry's own basis

Date

Filed

Basis

What it is

Start

2021-05-17

actual

When the study began enrolling

Primary completion

2027-08-07

estimated

The date the last participant is measured for the primary outcome — the readout window

Study completion

2028-04-30

estimated

The whole study's end, after follow-up

First posted

2021-04-23

actual

When this record first appeared on the registry

Results posted

—

When the sponsor posted results to the registry

Record updated

2026-03-30

actual

The sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation

Interventions

Drug: Tofersen — also filed as BIIB067, QALSODY
Drug: Placebo

Primary outcome

what the primary-completion date above is the date OF

Parts B and C: Percentage of Participants with Emergence of Clinically Manifest ALS Within 24 Months of Part B Baseline

measured Up to 24 months

Publications

PMID 35585374 — linked by the registry

Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.

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