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Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
What is ALS?
Amyotrophic lateral sclerosis, also called Lou Gehrig's disease, is a progressive disease of the motor neurons, the nerve cells that control voluntary muscles. As they die, people lose strength in the arms, legs, speech, swallowing, and eventually breathing, while thinking usually stays intact. Most people live two to five years after symptoms begin, though some live ten years or more, and younger age at onset tends to mean slower progression. About 5 to 10 percent of cases run in families; the rest are sporadic, with no known cause. Several genes are linked to ALS, including SOD1 and C9orf72, and testing for them matters because some trials and one approved drug target specific genes. Some people with ALS also develop frontotemporal dementia.
Types of ALS clinical trials
Disease slowing drug trials. Phase 3 studies of medicines meant to slow loss of function, measured with the standard ALS rating scale.
Gene targeted trials. Treatments for specific genetic forms, including SOD1 and C9orf72, and prevention studies in gene carriers without symptoms; see gene therapy clinical trials.
Cell therapy trials. Engineered immune or stem cells designed to protect motor neurons.
Biomarker and early detection studies. Blood, imaging, and breathing tests that track the disease and diagnose it sooner.
Care and quality of life studies. Breathing support, communication technology, and multidisciplinary clinic care.
Motor neuron and memory changes overlap with frontotemporal dementia. New to research? Read how informed consent works.
Is ALS hereditary?
Sometimes. About 5 to 10 percent of cases are familial, passed down through a gene change; the rest are sporadic. Genetic testing is increasingly offered to everyone with ALS, because some trials and one approved drug target specific genes.
Who can participate in ALS clinical trials?
Most drug trials enroll adults within a few years of symptom onset, with breathing capacity above a set level, because that is when there is most function to protect. Gene targeted trials require a confirmed variant, and prevention studies enroll gene carriers before symptoms. Biomarker and care studies accept people at any stage, and some enroll family members or healthy volunteers. Eligibility always varies by study.
Stages of ALS, and why they matter for trials
Early. Weakness in one region, such as a hand or speech. Most drug trials enroll here or within a set time from first symptoms.
Middle. Weakness spreads to several regions; help needed with some daily tasks. Some drug and symptom trials enroll.
Late. Most muscles affected, often with breathing support. Care and technology studies.
How to enroll in an ALS clinical trial
Identify your trial. Use the filters and match the stage or type and the treatment history named in study titles to your own.
Select your preferred location. Enter your city or state; large trials run at many sites, early phase studies at fewer centers.
Explore study details. Click "Learn More" for eligibility, prior treatment rules, and the visit schedule.
Complete the health profile. Click "Get started" to begin the 5-step application. Have three things ready: when your symptoms began and where, any genetic test results, and your most recent breathing test results.
Submit the application. A clinical trial coordinator reviews it and contacts you. Nothing is decided until informed consent, and participation is voluntary at every step.