Long-Term Evaluation of BIIB067 (Tofersen)

NCT ID: NCT03070119

Last Updated: 2025-08-29

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

139 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-03-08

Study Completion Date

2024-08-12

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 (tofersen) in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK), pharmacodynamic (PD), biomarker effects, and efficacy of BIIB067 administered to participants with ALS and a confirmed SOD1 mutation.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

BIIB067

Participants who have completed Parts A, B, or C of study 233AS101 will be placed in this arm.

Group Type EXPERIMENTAL

Tofersen

Intervention Type DRUG

Participants will receive a loading dose regimen followed by maintenance dosing.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Tofersen

Participants will receive a loading dose regimen followed by maintenance dosing.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

BIIB067 QALSODY

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Must have diagnosis of superoxide dismutase 1-amyotrophic lateral sclerosis (SOD1-ALS), and must have completed the End of Study Visit for either Parts A, B, or C of Study 233AS101 (NCT02623699) (i.e., were not withdrawn).
* If taking riluzole, participant must be receiving a stable dose for ≥30 days prior to Day 1.
* If taking edaravone, participant must have initiated edaravone ≥60 days (2 treatment cycles) prior to Day 1. Edaravone may not be administered on dosing days during this study.
* Medically able to undergo the study procedures, and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.
* For female participants of childbearing potential must agree to practice effective contraception during the study and be willing and able to continue contraception for 5 months after their last dose of study treatment.
* Participants from Study 233AS101 Parts A and B must have a washout ≥16 weeks between the last dose of study treatment received in Study 233AS101 and the first dose of BIIB067 received in the current Study 233AS102.

Exclusion Criteria

* History of allergies to a broad range of anesthetics.
* Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally and could place a participant at an increased risk for bleeding during or after a Lumbar Puncture (LP) procedure. These risks could include, but are not limited to, anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von Willebrand's disease, liver disease).
* Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter.
* Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy.
* Treatment with another investigational drug, biological agent (excluding BIIB067), or device within 1 month or 5 half-lives of study agent, whichever is longer.
* Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.
* Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl-bis(N4-methylthiosemicarbazone)) or pyrimethamine.
* Female participants who are pregnant or currently breastfeeding.
* Current enrollment in any other interventional study.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role collaborator

Biogen

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Medical Director

Role: STUDY_DIRECTOR

Biogen

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Research Site

Phoenix, Arizona, United States

Site Status

Research Site

La Jolla, California, United States

Site Status

Research Site

San Francisco, California, United States

Site Status

Research Site

Jacksonville, Florida, United States

Site Status

Research Site

Miami, Florida, United States

Site Status

Research Site

Orlando, Florida, United States

Site Status

Research Site

Atlanta, Georgia, United States

Site Status

Research Site

Chicago, Illinois, United States

Site Status

Research Site

Baltimore, Maryland, United States

Site Status

Research Site

Boston, Massachusetts, United States

Site Status

Research Site

Rochester, Minnesota, United States

Site Status

Research Site

St Louis, Missouri, United States

Site Status

Research Site

Lincoln, Nebraska, United States

Site Status

Research Site

Cleveland, Ohio, United States

Site Status

Research Site

Portland, Oregon, United States

Site Status

Research Site

Knoxville, Tennessee, United States

Site Status

Research Site

Leuven, , Belgium

Site Status

Research Site

Calgary, Alberta, Canada

Site Status

Research Site

Edmonton, Alberta, Canada

Site Status

Research Site

Toronto, Ontario, Canada

Site Status

Research Site

Montreal, Quebec, Canada

Site Status

Bispebjerg Hospital

Copenhagen, , Denmark

Site Status

Research Site

Clermont-Ferrand, Puy De Dome, France

Site Status

Research Site

Ulm, Baden-Wurttemberg, Germany

Site Status

Research Site

Torino, , Italy

Site Status

Research Site

Bunkyō City, , Japan

Site Status

Research Site

Kagoshima, , Japan

Site Status

Research Site

Shinjuku-ku, , Japan

Site Status

Research Site

Suita-shi, , Japan

Site Status

Research Site

Sheffield, South Yorkshire, United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Belgium Canada Denmark France Germany Italy Japan United Kingdom

References

Explore related publications, articles, or registry entries linked to this study.

Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chio A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Cochrane T, Chary S, Chew S, Zhu H, Wu F, Nestorov I, Graham D, Sun P, McNeill M, Fanning L, Ferguson TA, Fradette S; VALOR and OLE Working Group. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2022 Sep 22;387(12):1099-1110. doi: 10.1056/NEJMoa2204705.

Reference Type DERIVED
PMID: 36129998 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2016-003225-41

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

233AS102

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.