Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

NCT07294144 · ClinicalTrials.gov registry record · Phase 2

Tofersen in Non-SOD1 ALS

A Phase 2 study of ALS (Amyotrophic Lateral Sclerosis), sponsored by Washington University School of Medicine.

Recruiting
Registry status
Phase 2
Development phase
30
Enrollment target
1
Study location

NCT07294144 is a Phase 2 study of ALS (Amyotrophic Lateral Sclerosis) that is actively recruiting participants, run by Washington University School of Medicine. The registered enrollment target is 30 participants, below the 363-participant average among 6 other ALS (Amyotrophic Lateral Sclerosis) trials with a reported enrollment target (92% lower). The trial reports 1 study location across 1 state.

View on ClinicalTrials.gov ↗

View your shortlist →

The verdict

NCT07294144, a Phase 2 study of ALS (Amyotrophic Lateral Sclerosis), is actively recruiting participants, sponsored by Washington University School of Medicine.

RECRUITING
Registry status
Phase 2
Development phase
30 participants
Enrollment target
1
Study location

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.

Interventions

  • DRUG Tofersen

Study Locations (1)

Missouri

  • Washington University ALS Center - St Louis

Trial Details

FieldValue
Enrollment Target 30 participants
Start Date 2025-12-29
Est. Completion 2028-05
Phase Phase 2

What the Registry Record Tells You About NCT07294144

The ClinicalTrials.gov registry entry for NCT07294144 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 30 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 363-participant average among 6 other ALS (Amyotrophic Lateral Sclerosis) trials with a reported enrollment target (92% lower). The listed sponsor is Washington University School of Medicine, which has 1,502 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with ALS (Amyotrophic Lateral Sclerosis) appearing as the primary indexed condition, and to 1 intervention - of which Tofersen is the first listed.

NCT07294144 reports 1 study location spanning 1 distinct geographic area - top geographies include Missouri.

Frequently Asked Questions

What is clinical trial NCT07294144 about?

NCT07294144 is a clinical study titled "Tofersen in Non-SOD1 ALS". 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 l...

What is the current status of trial NCT07294144?

This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 30 participants. The study started on 2025-12-29. Estimated completion is 2028-05.

What conditions does trial NCT07294144 study?

This clinical trial studies the following conditions: ALS (Amyotrophic Lateral Sclerosis).

What interventions are being tested in trial NCT07294144?

The interventions under investigation include: Tofersen (DRUG).

Who is sponsoring clinical trial NCT07294144?

This trial is sponsored by Washington University School of Medicine, which has 1,502 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT07294144 being conducted?

This trial has 1 study location across Missouri. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Similar trials for ALS (Amyotrophic Lateral Sclerosis)

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.