ALS Clinical Trials | ALS Therapy Development Institute

Your participation in an ALS clinical trial provides researchers with the data they need to determine whether or not a potential treatment is safe and effective, and ultimately, whether or not it should be available as a medication to all those with ALS. The ALS Therapy Development Institute is unbiased in our listing of clinical trials and provides information on all those enrolling worldwide.

We encourage you to browse trials below and subscribe to our clinical trials email to receive monthly updates with changes to trials listed in this database.

Currently Enrolling Clinical Trials

List includes only interventional trials with locations in the US.

Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALS/FTD

Phase 2 - Recruiting

Enrollment: 18

Drug: Single group assignment

Type: Interventional

Open Label: Unknown

Countries: US

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Evaluation of MN-166 (Ibudilast) for 12 Months Followed by an Open-label Extension for 6 Months in Patients With ALS

Phase 2/3 - Recruiting

Enrollment: 230

Drug: Drug

Type: Interventional

Open Label: Yes

Countries: CA, Canada, US

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A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

Phase 3 - Recruiting

Enrollment: 77

Drug: Unknown

Type: Interventional

Open Label: Yes

Countries: Belgium, CA, Italy, Netherlands, Other, United Kingdom, US

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Study of Safety and of the Mechanism of BLZ945 in ALS Patients

Phase 2 - Recruiting

Enrollment: 56

Drug: Open label extension

Type: Interventional

Open Label: No

Countries: Finland, Sweden, US

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Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients

Phase 3 - Recruiting

Enrollment: 495

Drug: Drug - oral

Type: Interventional

Open Label: No

Countries: Belgium, Denmark, France, Germany, Greece, Israel, Italy, Norway, Other, Poland, Portugal, Russian Federation, Slovenia, Spain, Sweden, Ukraine, US

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

Phase 3 - Recruiting

Enrollment: 150

Drug: Unknown

Type: Interventional

Open Label: Yes

Countries: Australia, Belgium, Brazil, CA, France, Germany, Italy, Japan, Other, Poland, Spain, Sweden, United Kingdom, US

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A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)

Phase 2 - Recruiting

Enrollment: 63

Drug: Unknown

Type: Interventional

Open Label: No

Countries: Belgium, CA, Germany, Other, Sweden, US

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RAPA-501 Therapy for ALS

Phase 2/ 3 - Recruiting

Enrollment: 41

Drug: RAPA-501 Autologous T cells

Type: Interventional

Open Label: Unknown

Countries: US

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HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262

Phase 2/3 - By Invitation

Enrollment: 240

Drug: Unknown

Type: Interventional

Open Label: Yes

Countries: US, United States

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Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS

Phase 2 - Recruiting

Enrollment: 258

Drug: Unknown

Type: Interventional

Open Label: Yes

Countries: Australia, France, Poland, Spain, US

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Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial

Phase 2 - Recruiting

Enrollment: 60

Drug: Combination Antioxidants

Type: Interventional

Open Label: Unknown

Countries: US

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Adenosine 2A Receptor Antagonism and AIH in ALS

Phase 1/ 2 - Recruiting

Enrollment: 40

Drug: Unknown

Type: Interventional

Open Label: No

Countries: US

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Neurodegenerative Alzheimer's Disease and Amyotrophic Lateral Sclerosis (NADALS) Basket Trial

Phase 1/2 - Recruiting

Enrollment: 20

Drug: Unknown

Type: Interventional

Open Label: Unknown

Countries: US

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CNS10-NPC-GDNF Delivered to the Motor Cortex for ALS

Phase 1 - Recruiting

Enrollment: 16

Drug: Unknown

Type: Interventional

Open Label: No

Countries: US

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Intermediate Expanded Access Protocol (EAP) CNMAu8.EAP02

N/A - Available

Enrollment: 300

Drug: Unknown

Type: Expanded Access

Open Label: Unknown

Countries: US

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Intermediate-Sized Expanded Access Study

N/A - Available

Enrollment: 70

Drug: Unknown

Type: Expanded Access

Open Label: No

Countries: US

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HEALEY ALS Platform Trial - Regimen G DNL343

Phase 2/3 - By Invitation

Enrollment: 240

Drug: Unknown

Type: Interventional

Open Label: Yes

Countries: US, United States

Learn More

Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALS/FTD

Phase 2 - Recruiting

Enrollment: 18

Drug: Single group assignment

Type: Interventional

Open Label: Unknown

Countries: US

Learn More

Evaluation of MN-166 (Ibudilast) for 12 Months Followed by an Open-label Extension for 6 Months in Patients With ALS

Phase 2/3 - Recruiting

Enrollment: 230

Drug: Drug

Type: Interventional

Open Label: Yes

Countries: CA, Canada, US

Learn More

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

Phase 3 - Recruiting

Enrollment: 77

Drug: Unknown

Type: Interventional

Open Label: Yes

Countries: Belgium, CA, Italy, Netherlands, Other, United Kingdom, US

Learn More

Study of Safety and of the Mechanism of BLZ945 in ALS Patients

Phase 2 - Recruiting

Enrollment: 56

Drug: Open label extension

Type: Interventional

Open Label: No

Countries: Finland, Sweden, US

Learn More

Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients

Phase 3 - Recruiting

Enrollment: 495

Drug: Drug - oral

Type: Interventional

Open Label: No

Countries: Belgium, Denmark, France, Germany, Greece, Israel, Italy, Norway, Other, Poland, Portugal, Russian Federation, Slovenia, Spain, Sweden, Ukraine, US

Learn More