US Interventional/Drug Trials Only

Updated August 29, 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 ranolazine 2
  • placebo controlled trial evaluating two doses of ranolazine
  • sites in CA, FL, KS, MO, OH
 Swathy Chandrashekhar, MBBS
3 digoxin 2
  • no placebo
  • site in MA
 Massachusetts General Hospital
4 Vit E, N-Ac cysteine, L-cystine, Nicotinamide, Taurursodiol 2
  • no placebo
  • site in TX
 Dallas VA Medical Center
5 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
6 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
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

 UniQure Biopharma B.V.
8 Psilocybin 1
  • no placebo
  • site in MD
 Johns Hopkins University
9 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 
 Amylyx Pharmaceuticals Inc.
10 prosetin 1
  • placebo controlled trial lasting 14 days followed by open label extension
  • oral drug designed to target MAP4 kinase
  • site in MA
 ProJenX
11 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
 Mayo Clinic
12 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