Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a rapidly progressive fatal neurodegenerative disease affecting both upper and lower motor neurons. ALS clinical trials have historically had a high failure rate due to disease heterogeneity, rapid progression, and lack of validated biomarkers — but recent approvals of tofersen (for SOD1-ALS) and the conditional approval of AMX0035 (subsequently withdrawn from the EU) represent important steps forward. The discovery that 10–15% of ALS patients carry identifiable genetic mutations (SOD1, C9orf72, FUS, TDP-43) has enabled genotype-targeted trials.
Active trials investigate antisense oligonucleotides (ASOs) targeting SOD1, C9orf72, and FUS mutations; stem cell therapies (NurOwn); the HEALEY ALS Platform Trial (testing multiple agents simultaneously); brain-computer interfaces for communication; and biomarker studies using neurofilament light chain (NfL) to track disease activity and predict outcomes. Gene therapy approaches for SOD1-ALS are in early-phase development.
ALS trial eligibility typically requires confirmed ALS diagnosis per El Escorial criteria, symptom onset within 1–2 years, adequate respiratory function (FVC ≥60%), and in genetically targeted trials, confirmed mutation status.
Frequently Asked Questions — randomized controlled trials Clinical Trials
How many clinical trials are currently recruiting for randomized controlled trials?
ClinicalMetric currently tracks 2 actively recruiting clinical trials for randomized controlled trials, sourced in real time from ClinicalTrials.gov. The total number of registered studies—including those not yet enrolling or in active follow-up—is 2. Trial availability changes daily as new studies open enrollment and existing ones reach capacity.
What trial phases are available for randomized controlled trials?
randomized controlled trials research spans Phase 3 (1 trial). Phase 1 studies evaluate safety and dosing in small groups, Phase 2 studies assess preliminary efficacy in 100–300 participants, and Phase 3 trials compare the new treatment against the standard of care in 300–3,000+ patients. Phase 4 post-approval studies monitor long-term outcomes in real-world populations.
How do I find out if I qualify for a randomized controlled trials clinical trial?
Eligibility criteria for randomized controlled trials trials vary by study and typically specify age range, disease stage or severity, prior treatment history, and specific diagnostic or laboratory parameters. Each listing on ClinicalMetric links to the full protocol on ClinicalTrials.gov, where inclusion and exclusion criteria are documented. Contact the sponsoring site's research coordinator directly to confirm your eligibility—your treating physician or specialist can also help identify the most appropriate trial based on your medical history and current treatment status.
Top Sponsors
Hospital for Special Surgery, New York 1 trial
Rennes University Hospital 1 trial
Recruiting Clinical Trials
ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional.
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Last Reviewed: July 2026 ·
Data Methodology