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ClinicalTrials.gov public records Last synced Jul 22, 2026, 6:34 PM EDT

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Showing 1–20 of 20 matching trials from the live ClinicalTrials.gov search.
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Filters: "SOD1" Condition: ALS Clear all
Recruiting No phase listed Observational

Amyotrophic Lateral Sclerosis (ALS) Families Project

NCT03865420
View directory record Official record
Conditions
ALS
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 105 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced Jul 22, 2026, 6:34 PM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)

NCT03506425
View directory record Official record
Conditions
ALS
Interventions
Triheptanoin
Drug
Lead sponsor
Richard Bedlack, M.D., Ph.D.
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 23, 2019 · Synced Jul 22, 2026, 6:34 PM EDT
Not listed No phase listed Observational

Rehabilitation in SOD1 ALS Treated With Tofersen

NCT05725759
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Familial Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Motor Neuron Disease, Familial
Interventions
Rehabilitation
Behavioral
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 29, 2024 · Synced Jul 22, 2026, 6:34 PM EDT
Withdrawn Phase 2 Interventional

A Multicenter, Double-Blind Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With ALS

NCT00561366
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Placebo, Arimoclomol
Drug
Lead sponsor
CytRx
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
31
States / cities
Pacific Palisades, California • San Francisco, California • Denver, Colorado + 26 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2012 · Synced Jul 22, 2026, 6:34 PM EDT
Approved For Marketing No phase listed Expanded access

Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

NCT04972487
View directory record Official record
Conditions
Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
Interventions
Tofersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years and older
U.S. locations
19
States / cities
Anchorage, Alaska • Los Angeles, California • Orange, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2024 · Synced Jul 22, 2026, 6:34 PM EDT
Recruiting No phase listed Observational

The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study

NCT00317616
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2037
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Mar 19, 2026 · Synced Jul 22, 2026, 6:34 PM EDT
Completed Phase 3 Interventional Results available

An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)

NCT02623699
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Tofersen, Placebo
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years and older
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
20
States / cities
Phoenix, Arizona • La Jolla, California • San Francisco, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2023 · Synced Jul 22, 2026, 6:34 PM EDT
Recruiting Phase 2 Interventional

Tofersen in Non-SOD1 ALS

NCT07294144
View directory record Official record
Conditions
ALS (Amyotrophic Lateral Sclerosis)
Interventions
Tofersen
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
3
States / cities
Atlanta, Georgia • Boston, Massachusetts • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Jul 22, 2026, 6:34 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Analysis of Post-Translational Modifications of a Critical Protein Implicated in Amyotrophic Lateral Sclerosis

NCT02228915
View directory record Official record
Conditions
ALS
Interventions
Not listed
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
18 Years to 99 Years
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2018
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 16, 2018 · Synced Jul 22, 2026, 6:34 PM EDT
Completed Phase 2 Interventional

GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS)

NCT01854294
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
GM604, Placebo comparator
Drug
Lead sponsor
Genervon Biopharmaceuticals, LLC
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 26, 2021 · Synced Jul 22, 2026, 6:34 PM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders

NCT01459302
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, PLS, Motor Neuron Disease, Lou Gehrigs Disease, Familial Disease, Amyotrophic Lateral Sclerosis, Sporadic, Muscular Dystrophy, Miyoshi Myopathy, Amyotrophic Lateral Sclerosis With Dementia
Interventions
Not listed
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2024
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced Jul 22, 2026, 6:34 PM EDT
Suspended No phase listed Observational Accepts healthy volunteers

SOD1 Kinetics Measurements in ALS Patients

NCT03449212
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Familial, Amyotrophic Lateral Sclerosis, Sporadic
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
86 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2026
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 24, 2024 · Synced Jul 22, 2026, 6:34 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

NCT06100276
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
AMT-162
Drug
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
11
States / cities
Phoenix, Arizona • Irvine, California • San Francisco, California + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Jul 22, 2026, 6:34 PM EDT
Recruiting No phase listed Observational

A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)

NCT07259980
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Tofersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years and older
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2033
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Jul 22, 2026, 6:34 PM EDT
Completed Phase 1Phase 2 Interventional Results available

SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS)

NCT01083667
View directory record Official record
Conditions
Familial Amyotrophic Lateral Sclerosis
Interventions
Pyrimethamine
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
2
States / cities
New York, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 18, 2017 · Synced Jul 22, 2026, 6:34 PM EDT
Completed Phase 2Phase 3 Interventional

Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis

NCT00706147
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Arimoclomol, Placebo
Drug
Lead sponsor
University of Miami
Other
Eligibility
18 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
2
States / cities
Miami, Florida • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 10, 2019 · Synced Jul 22, 2026, 6:34 PM EDT
Recruiting Phase 1 Interventional

A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)

NCT07290062
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
INS1202
Genetic
Lead sponsor
Insmed Gene Therapy LLC
Industry
Eligibility
18 Years to 79 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
5
States / cities
La Jolla, California • Palo Alto, California • Columbia, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 5, 2026 · Synced Jul 22, 2026, 6:34 PM EDT
Completed Phase 1 Interventional

Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations

NCT01041222
View directory record Official record
Conditions
Familial Amyotrophic Lateral Sclerosis
Interventions
ISIS 333611
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
4
States / cities
Baltimore, Maryland • Charlestown, Massachusetts • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2012 · Synced Jul 22, 2026, 6:34 PM EDT
Active, not recruiting Phase 3 Interventional

A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

NCT04856982
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation
Interventions
Tofersen, Placebo
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years and older
Enrollment
158 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
12
States / cities
Scottsdale, Arizona • La Jolla, California • San Francisco, California + 9 more
Source: ClinicalTrials.gov public record
Updated Mar 29, 2026 · Synced Jul 22, 2026, 6:34 PM EDT
Completed Phase 3 Interventional Results available

Long-Term Evaluation of BIIB067 (Tofersen)

NCT03070119
View directory record Official record
Conditions
ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation
Interventions
Tofersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years and older
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
16
States / cities
Phoenix, Arizona • La Jolla, California • San Francisco, California + 13 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2025 · Synced Jul 22, 2026, 6:34 PM EDT