Actively Recruiting ALS Clinical Trials

• antisense oligonucleotide treatment for people carrying a mutation in FUS gene
• recruiting in CA, MD, MA, MO, NY, OH, UT

• 33% chance of placebo
• trial volunteers will be separated into 3 groups, a high dose with riluzole, a low dose with riluzole, or placebo with riluzole
• recruiting in AL, CA, KY, MD, MA, VA

• biomarkers will be monitored over time and tofersen or placebo will be administered if specific biomarker changes occur
• antisense oligonucleotide treatment for people carrying specific SOD1 mutations, but no current symptoms of ALS
• recruiting in AZ, CA, FL, GA, IL, MD, MA, MO, NY, TX

DNL 343

Regimen G

Healey Platform Trial

• 25% chance of placebo for 6 months followed by active treatment extension for all participants
• single dose is being tested
• recruiting in AZ, AR, CA, CO, CT, DC, FL, GA, ID, IL, IN, IA, KS, KY, MD, MA, MI, MN, MO, NE, NH, NJ, NY, NC, OH, OR, PA, TN, TX, UT, VA, WA, WI
• *many sites have completed enrollment- please reach out to individual sites for more info

• 50% chance of placebo for 12 months followed by an open label extension for 6 months
• recruiting in CA, FL, GA, IN, KS, MD, MN, NY, NC, OR, PA, VA

• no placebo
• autologous T cells are administered intravenously for 6 months followed by an additional 6 months of observation
• recruiting in MA, NJ

• placebo-controlled trial
• drug is delivered intravenously
• trial is for people carrying SOD1 mutation or sporadic ALS
• recruiting in CA

• no placebo
• multiple groups or “cohorts” with cohorts 1-4 all receiving treatment and PET imaging, while in cohort 5 participants will be split into two groups that will either receive PET imaging or contribute to spinal fluid analysis
• recruiting in CA, CT, MA

• no placebo
• recruiting in TX

• crossover study: all trial volunteers will receive placebo and all trial volunteers will receive active treatment at different timepoints
• recruiting in FL

• no placebo
• includes people living with different neurodegenerative diseases including ALS
• recruiting in MA

• placebo controlled trial
• umbilical cord derived T regulatory cells delivered intravenously
• recruiting in NY, TX

• no placebo
• cells that produce GDNF (a growth factor) will be transplanted into motor cortex of brain
• recruiting in CA

• Expanded Access Program for people that are not eligible for trials
• CNM-Au8 will be administered for up to  96 weeks orally or by feeding tube
• sites in AZ, CA, CT, FL, IL, KS, MI, NE, NC, OR, PA, TX, 

• Expanded Access Program for people that are not eligible for trials
• Autologous hybrid TREG/Th2 cells delivered intravenously
• sites in MA, NJ

• Expanded Access Program for people that are not eligible for trials
• pridopidine will be administered twice daily by mouth or feeding tube for 2 years
• sites not yet listed, but contact information is available on clinicaltrials.gov