US Interventional/Drug Trials Only

Updated April 15, 2025

 

ALS TDI updates this list of ALS clinical trials monthly.

 

  Drug Phase Study Design Sponsor
1 ION363 (ulefnersen) 3
  • antisense oligonucleotide treatment for people carrying a mutation in FUS gene
  • sites in CA, MD, MA, MO, NY, OH, UT
 Ionis Pharmaceuticals, Inc.
2 MN-166 (ibudilast) 2/3
  • 50% chance of placebo for 12 months followed by an open label extension for 6 months
  • sites in CA, FL, GA, IN, KS, MD, MN, NY, NC, OR, PA, VA
 MediciNova
3 RAPA-501 2/3
  • no placebo
  • autologous hybrid TREG/Th2 cells delivered intravenously
  • EAP also availible
  • sites in MA,NJ
 Rapa Therapeutics LLC
4 Vit E, N-Ac cysteine, L-cystine, Nicotinamide, Taurursodiol 2
  • no placebo
  • site in TX
 Dallas VA Medical Center
5 VHB937 2
  • Monoclonal antibody meant to improve microglial function
  • Placebo controlled trial, randomized 2:1
  • 40-week treatment period followed by an open label extension
  • Site in TX
 Novartis Pharmaceuticals
6 AMT-162 1/2
  • no placebo
  • viral gene therapy for SOD1-related ALS
  • one-time intrathecal injection, with up to five years of follow-up
  • participants cannot be on tofersen
  • sites in CA, IL, MA, NY, PA, FL, KS, MN

 UniQure Biopharma B.V.
7 istradefylline and acute intermittent hypoxia 1/2
  • crossover study: all trial volunteers will receive placebo and all trial volunteers will receive active treatment at different timepoints
  • site in FL
 University of Florida
8 CNS10-NPC-GDNF 1
  • no placebo
  • cells that produce GDNF (a growth factor) will be transplanted into motor cortex of brain
  • site in CA
 Cedars-Sinai Medical Center
9 FC-12738 (retro enversio thymopentin) 1
  • placebo controlled trial
  • testing a potential ALS treatment in healthy volunteers ONLY
  • site in NJ
 Neurodegenerative Disease Research Inc
10 RAPA-501 EAP
  • Expanded Access Program for people that are not eligible for trials
  • Autologous hybrid TREG/Th2 cells delivered intravenously
  • sites in MA, NJ
 Rapa Therapeutics LLC
11 CNM-Au8 EAP
  • 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, 
 Clene Nanomedicine
12 Pridopidine EAP
  • 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 in AZ, CA, CO, CT, DC, FL, ID, IL, IA, MA, MN, MO, NJ, NY, NC, OH, TX, VA, WV, WI, and Puerto Rico
 Prilenia