NCT07294144

Tofersen in Non-SOD1 ALS

A Study to Evaluate the Biological Effect of Tofersen in Adults With Amyotrophic Lateral Sclerosis Without Mutations in SOD1

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Washington University School of Medicine · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Type	Name	Description
DRUG	Tofersen	Tofersen 100 mg administered intrathecally.

Timeline

• Start date: 2025-12-29
• Primary completion: 2027-01-01
• Completion: 2028-05-01
• First posted: 2025-12-19
• Last updated: 2026-02-09

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study