Tofersen in Non-SOD1 ALS

Part of paid clinical trials in Atlanta, Georgia.

Sponsor
Washington University School of Medicine
Study ID
NCT07294144
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • ALS (Amyotrophic Lateral Sclerosis)

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Tofersen — DRUG
    Tofersen 100 mg administered intrathecally.

Study Details

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.

Key Dates

Start date
Dec 29, 2025
Status verified
Jun 2026
Primary completion
Jan 31, 2027
Completion
May 31, 2028

Study Design

Enrollment
30 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Tofersen

Primary Outcome Measure

Proportion of participants with ≥30% reduction in plasma NfL [ Time Frame: From Baseline to Week 28 ]

Locations (2)

FacilityCityStateZIPSite coordinators
Emory UniversityAtlantaGeorgia30322
Karon Simmons
[email protected]
404-712-4182
Jonathan Glass, MD (PRINCIPAL_INVESTIGATOR)
Washington University ALS CenterSt LouisMissouri63110
ALS Clinical Research Team
[email protected]
1-844-257-2273
Timothy M Miller, MD, PhD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Atlanta, GA

By research site
Emory University· Atlanta, GAWashington University ALS Center· St Louis, MO

Related Studies