Tofersen in Non-SOD1 ALS (NCT07294144)
A Study to Evaluate the Biological Effect of Tofersen in Adults With Amyotrophic Lateral Sclerosis Without Mutations in SOD1
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Emory University — Atlanta, Georgia
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (3 Sites)
- Emory University — Atlanta, Georgia
- Massachusetts General Hospital — Boston, Massachusetts
- Washington University ALS Center — St Louis, Missouri
📞 Contact the Study Coordinator
Phone: (404) 712-4182
Email: kmsimm2@emory.edu
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in ALS (Amyotrophic Lateral Sclerosis)
- T-regulatory Cells in ALS — Boston, Massachusetts
- Augmented Reality BCI Longitudinal Study for Persons With ALS, Stroke, TBI and SCI Utilizing Cognixion + Apple Vision Pro — Santa Barbara, California
- A Study of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis — Leuven,
- Augmented Reality BCI Longitudinal Study for Persons With Late Stage ALS — Santa Barbara, California
- Tongue-strengthening Exercises in People With ALS. — Davie, Florida
Explore by condition: All ALS (Amyotrophic Lateral Sclerosis) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.