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Tofersen in Non-SOD1 ALS

NCT07294144 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2026-02-09

No results posted yet for this study

Summary

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.

Conditions

  • ALS (Amyotrophic Lateral Sclerosis)

Interventions

DRUG

Tofersen

Tofersen 100 mg administered intrathecally.

Sponsors & Collaborators

  • Biogen

    collaborator INDUSTRY

  • Washington University School of Medicine

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-12-29
Primary Completion
2027-01-31
Completion
2028-05-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07294144 on ClinicalTrials.gov