Find Recruiting Clinical Trials for ALS (Amyotrophic Lateral Sclerosis)

ALS clinical trials span several categories. Genetic-targeted trials are the most targeted: tofersen (Qalsody) is approved for SOD1-ALS and expanded access/continuation programs exist; next-generation SOD1-targeting agents are in trials; C9orf72-targeting ASOs are in early-phase trials for C9-positive patients; FUS-targeting approaches are in development. Platform trials — particularly the HEALEY ALS Platform Trial at MGH, which tests multiple agents simultaneously under a master protocol — offer access to multiple investigational agents at a single site. Neuroprotective and anti-neuroinflammatory trials test agents modulating TDP-43 pathology, microglial activation, oxidative stress (NurOwn, SLS-005/trehalose), and mitochondrial function. Symptomatic and quality-of-life trials address bulbar symptoms, spasticity, nutritional support, and respiratory management. Biomarker and natural history studies are also valuable — they provide neurofilament measurements, genetic testing, and contribute to the evidence base while sometimes providing early access to trial sites. Patients should pursue genetic testing immediately, as it unlocks genetic-targeted trial options.

Yes — genetic testing is one of the most important steps any ALS patient can take. SOD1 mutations are present in approximately 20% of familial ALS and 2–3% of sporadic ALS — patients with confirmed SOD1 mutations are eligible for tofersen (approved) and trials of next-generation SOD1-targeting antisense oligonucleotides. C9orf72 hexanucleotide repeat expansion is the single most common genetic cause of ALS — present in approximately 40% of familial and 5–10% of sporadic cases — and C9orf72-targeting ASO trials specifically require this mutation. FUS, TDP-43 (TARDBP), UBQLN2, and other mutations also have dedicated trial programs emerging. Genetic testing should include a comprehensive ALS gene panel (SOD1, C9orf72, FUS, TARDBP, UBQLN2, OPTN, TBK1, NEK1, and others) from a certified laboratory. Testing is increasingly available through sponsored programs (ATLAS study by Biogen provides free genetic testing and counseling for ALS patients). Even patients with sporadic ALS and no family history should be tested, as de novo mutations occur. Genetic counseling is strongly recommended alongside testing.

FVC (forced vital capacity) — expressed as a percentage of the predicted value for your age, height, and sex — is one of the most commonly used eligibility cutoffs in ALS trials, and understanding your FVC is essential for trial planning. FVC is measured by a simple breathing test (spirometry) and reflects how much air you can forcefully exhale. In ALS, respiratory muscle weakness causes FVC to decline over time. Most ALS clinical trials require FVC ≥50% predicted at screening — some require ≥60%. This threshold exists because patients with more severely compromised respiratory function have reduced life expectancy, higher risk of respiratory complications during trial participation, and may not be able to tolerate study visits or procedures. Patients with FVC below the threshold are often excluded not because the treatment wouldn't help, but because the trial design cannot safely accommodate them. Critically, FVC can decline relatively quickly — patients who are at or near threshold should prioritize trial screening promptly, before FVC drops below the cutoff. Some compassionate use and expanded access programs have more flexible FVC requirements.

The HEALEY ALS Platform Trial is a groundbreaking master protocol trial based at Massachusetts General Hospital (MGH) in Boston, with partner sites across the United States and internationally. Rather than running separate trials for each investigational agent, the HEALEY platform tests multiple agents simultaneously under a single trial infrastructure — reducing the time and burden of traditional trial design. Participants are randomized across multiple treatment regimens (called "regimens") that are added, modified, or removed as evidence accumulates. This design accelerates drug development and allows more patients to access investigational agents at once. Eligibility for the HEALEY platform generally requires: confirmed ALS diagnosis by El Escorial criteria, FVC ≥60% at screening, ability to travel to a participating site, and willingness to undergo lumbar puncture (CSF collection) for some regimens. To find out if you are eligible and which regimens are currently enrolling, visit the HEALEY ALS Platform Trial website at healeyplatform.org or ask your neurologist for a referral to an ALS Center of Excellence. Participating sites include major academic ALS centers — early contact is advised as enrollment windows for individual regimens fill quickly.