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Study identifier: NCT07294144 Synced from ClinicalTrials.gov · August 04, 2026
● Recruiting

Tofersen in Non-SOD1 ALS

Condition: ALS (Amyotrophic Lateral Sclerosis)  ·  Sponsor: Washington University School of Medicine

PhasePhase 2
Planned participants30
Who can joinAll sexes, 18 Years to no upper limit
Healthy volunteersNo

About this study

The goal of this clinical trial is to evaluate whether tofersen is safe and effective in adults with non-SOD1 ALS. Tofersen is currently approved by the U.S. Food and Drug Administration to treat SOD1-ALS. The main questions it aims to answer are: * Does tofersen lower the levels of neurofilament light chain (NfL) in the blood and CSF of adult participants with non-SOD1 ALS? * Is tofersen safe and tolerable for adult participants with non-SOD1 ALS? * Does tofersen affect other measurements such as clinical outcomes and quality-of-life measures in participants with non-SOD1 ALS? Participants will : * Receive 100mg tofersen via lumbar puncture for 24 weeks. The doses are at the following time points: Weeks 0, 2, 4, 8, 12, 16, 20, and 24. * Complete 2 follow-up visits following the end of the dosing period at Weeks 28 and 32. * Complete a variety of questionnaires and outcome measurements such as strength and breathing testing.

This description comes directly from the study's public registry record.

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Contact details are listed per location below or on the registry record.

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Locations (3)

Emory UniversityAtlanta, Georgia, United StatesRecruiting
Massachusetts General HospitalBoston, Massachusetts, United StatesRecruiting
Washington University ALS CenterSt Louis, Missouri, United StatesRecruiting

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