Amyotrophic Lateral Sclerosis (ALS) Clinical Trials

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease characterized by degeneration of both upper motor neurons (in the brain cortex) and lower motor neurons (in the spinal cord), leading to weakness and paralysis of voluntary muscles. Sensation is typically preserved, but up to about half of people develop some change in thinking, language or behavior, and a smaller share develop frontotemporal dementia. The disease typically begins with focal weakness in one limb or in speech and swallowing muscles, then progresses to generalized weakness.

The most common symptoms of Amyotrophic Lateral Sclerosis include Muscle weakness in arms, legs, or both, often starting in one limb, Muscle twitching (fasciculations) visible under the skin, Progressive loss of strength and muscle control, Difficulty speaking, slurred speech, Difficulty swallowing, among others. About 5,000 new cases per year in the U.S.; CDC's National ALS Registry estimates about 33,000 Americans were living with ALS in 2022, a number projected to pass 36,000 by 2030. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Amyotrophic Lateral Sclerosis from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. ALS research is exceptionally active, with multiple trials evaluating neuroprotective agents, antisense oligonucleotides targeting disease genes, and emerging immune-modulating approaches. On September 22, 2026, Ionis reported that the Phase 3 FUSION trial of ulefnersen, an experimental drug for ALS caused by changes in the FUS gene, met its main goal, though the drug is not FDA approved.

Yes. As of October 21, 2026, 3 FDA-approved medicines have a current label that names Amyotrophic Lateral Sclerosis: Qalsody (tofersen), first approved in 2023; Radicava (edaravone), first approved in 2017; and Rilutek (riluzole), first approved in 1995. Corticosteroids and immune globulins count once each.

Amyotrophic Lateral Sclerosis is caused by variants in any of many genes, most often C9orf72, SOD1, TARDBP and FUS. About 90-95% of ALS is sporadic; among familial cases, C9orf72 repeat expansions account for 30-40% and SOD1 for 15-20%, and a cause is found in about 60%.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it. About 5,000 new cases per year in the U.S.; CDC's National ALS Registry estimates about 33,000 Americans were living with ALS in 2022, a number projected to pass 36,000 by 2030. A multigene panel identifies the cause in most families.

Yes. Amyotrophic Lateral Sclerosis can be caused by variants in many different genes (C9orf72, SOD1, TARDBP and FUS are among the most common), so testing is usually a multigene panel or exome sequencing rather than one gene at a time. About 90-95% of ALS is sporadic; among familial cases, C9orf72 repeat expansions account for 30-40% and SOD1 for 15-20%, and a cause is found in about 60%.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it.

Knowing the exact gene matters: it decides which trials a patient qualifies for and which relatives should be tested. The labs currently offering the test are listed in the Genetic Testing section of this page, pulled live from the NIH Genetic Testing Registry. A genetic counselor can help interpret the result and decide which relatives should be tested.

Eligibility for Amyotrophic Lateral Sclerosis clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Amyotrophic Lateral Sclerosis clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.