Tofersen

DRUG8 trials

Sponsors

Biogen, Washington University School of Medicine

Conditions

ALS (Amyotrophic Lateral Sclerosis)ALS Caused by Superoxide Dismutase 1 (SOD1) MutationAmyotrophic Lateral SclerosisAmyotrophic Lateral Sclerosis Associated With a SOD1 Gene MutationHealthy VolunteersSuperoxide Dismutase 1-Amyotropic Lateral Sclerosis

Phase 1

A Study to Evaluate Safety, Tolerability, and Distribution of a Microdose of Radiolabeled BIIB067 (99mTc-MAG3-BIIB067) Co-administered With BIIB067 (Tofersen) to Healthy Adults

CompletedNCT03764488

BiogenHealthy Volunteers

Start: 2018-12-20End: 2021-07-10Updated: 2023-06-22

Phase 2

Tofersen in Non-SOD1 ALS

RecruitingNCT07294144

Washington University School of MedicineALS (Amyotrophic Lateral Sclerosis)

Start: 2025-12-29End: 2028-05-01Target: 30Updated: 2026-06-25

Phase 3

An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)

CompletedNCT02623699

BiogenAmyotrophic Lateral Sclerosis

Start: 2016-01-20End: 2021-07-16Updated: 2023-07-28

Long-Term Evaluation of BIIB067 (Tofersen)

CompletedNCT03070119

BiogenALS Caused by Superoxide Dismutase 1 (SOD1) Mutation

Start: 2017-03-08End: 2024-08-12Updated: 2025-08-29

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

Active, not recruitingNCT04856982

BiogenAmyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation

Start: 2021-05-17End: 2028-04-30Updated: 2026-03-30

Phase 4

A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)

Active, not recruitingNCT07223723

BiogenAmyotrophic Lateral Sclerosis

Start: 2025-12-11End: 2027-05-26Target: 12Updated: 2026-08-13

Unknown Phase

Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

BiogenSuperoxide Dismutase 1-Amyotropic Lateral Sclerosis

Updated: 2024-06-10

A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)

RecruitingNCT07259980

BiogenAmyotrophic Lateral Sclerosis

Start: 2026-01-02End: 2033-12-30Target: 125Updated: 2026-04-09

Related Papers

Antisense Oligonucleotide Tofersen Distribution in the Central Nervous System of SOD1-ALS Autopsy Tissue Donors.

2026-07-06

Long-Term Tofersen in <i>SOD1</i> Amyotrophic Lateral Sclerosis

JAMA Neurology2025-12-22

Recent advances in RNA-targeting therapy for neurological diseases

Neural Regeneration Research2023-04-103 citations

Trial of Antisense Oligonucleotide Tofersen for <i>SOD1</i> ALS

New England Journal of Medicine2022-09-21758 citations

Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study.

2022-05-18124 citations

Paving the Way Toward Meaningful Trials in Ataxias: An Ataxia Global Initiative Perspective

Movement Disorders2022-04-2742 citations

Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for <i>SOD1</i> ALS

New England Journal of Medicine2020-07-08516 citations