US Interventional/Drug Trials Only

Updated September 19, 2025

 

ALS TDI updates this list of ALS clinical trials monthly.

 

  Drug Phase Study Design Sponsor
1 RAPA-501 2/3
  • no placebo
  • autologous hybrid TREG/Th2 cells delivered intravenously
  • site in MA
Rapa Therapeutics LLC
2 STRO4 2
  • no placebo
  • autologous bone-marrow-derived mesenchymal stem cells delivered intravenously
  • site in TX
New England Cell Therapeutics, Inc.
3 digoxin 2
  • no placebo
  • sites in MA, FL
Massachusetts General Hospital
4 Vit E, N-Ac cysteine, L-cystine, Nicotinamide, Taurursodiol 2
  • no placebo
  • site in TX
Dallas VA Medical Center
5 ranolazine 2
  • placebo controlled trial evaluating two doses of ranolazine
  • sites in CA, KS, OH
Swathy Chandrashekhar, MBBS
6 VHB937 2
  • 33% chance of placebo for 40 weeks followed by an open label extension
  • intravenous delivery of an antibody designed to activate TREM2 protein
  • sites in CA, FL, GA, KS, MA, NE, NY, OH, TE, TX
Novartis Pharmaceuticals
7 AMT-162 1/2
  • no placebo
  • one-time gene therapy for SOD1-related ALS
  • up to five years of follow-up
  • sites in CA, MA, NY, MN, FL, GA, KS,PA 

UniQure Biopharma B.V.
8 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
  • sites in FL
University of Florida
9 prosetin 1
  • placebo controlled trial lasting 14 days followed by open label extension
  • oral drug designed to target MAP4 kinase
  • site in MA
ProJenX
10 Psilocybin 1
  • no placebo
  • site in MD
Johns Hopkins University
11 AMX0114 1
  • 25% chance of placebo
  • antisense oligoneucleotide not limited to genetic ALS and designed to target calpain 2
  • intrathecal delivery of up to 4 doses
  • sites in CA, DC, FL, MA, TN, TX, MN
Amylyx Pharmaceuticals Inc.
12 MN-166 (ibudilast) EAP
  • Expanded Access Programfor people that are not eligible for trials
  • Contact WideTrial to join interest list to participate and see additional contact info here
  • Site in FL, AZ, MN
Mayo Clinic
13 RAPA-501 EAP
  • Expanded Access Program for people that are not eligible for trials
  • Autologous hybrid TREG/Th2 cells delivered intravenously
  • sites in AZ, CA, GA, IA, MA, MN, NJ, OR
Rapa Therapeutics LLC