US Interventional/Drug Trials Only

Updated December 5, 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 Usnoflast 2
  • 36 weeks study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of Usnoflast administered to adult subjects with Amyotrophic Lateral Sclerosis
  • Followed by 16 weeks open label extension study
  • sites in TX
Zydus Therapeutics Inc.
4 PHENOGENE-1A (Cromolyn) 2
  • Multifunctional therapy based on a repurposed drug, formulated with novel targeted delivery technology
  • This approach is designed to achieve therapeutic concentrations in both blood and brain, where it acts on key mechanisms involved in neuroinflammation and neurodegeneration
  • sites in CA, FL, NY
PhenoNet, Inc.
5 COYA 302 2
  • Combination therapy with a dual immunomodulatory mechanism of action intended to enhance the anti-inflammatory function of regulatory T cells (Tregs) and suppress the inflammation produced by activated monocytes and macrophages
  • sites in GA, MA, MO, NE, TX, 
Coya Therapeutics
6 Vit E, N-Ac cysteine, L-cystine, Nicotinamide, Taurursodiol 2
  • no placebo
  • site in TX
Dallas VA Medical Center
7 ranolazine 2
  • placebo controlled trial evaluating two doses of ranolazine
  • sites in CA, FL, KS, OH, MO
Swathy Chandrashekhar, MBBS
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, MN, PA, TN, TX
Amylyx Pharmaceuticals Inc.
12 acamprosate 1
  • no placebo
  • recruiting people with C9orf72-related ALS 
  • site in MD
National Institute of Neurological Disorders and Stroke (NINDS)
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
14 SPG302 EAP
  • Expanded Access Program for people that are not eligible for trials
  • Once a day oral pill that aims to restore synapses
  • Sites in IL, MA, NJ
Spinogenix
15 MN-166 (ibudilast) EAP
  • Expanded Access Program for people that are not eligible for trials
  • Contact WideTrial to join interest list to participate and see additional contact info here
  • Site in AZ, CA, CT, FL, IN, MN, NC, TN, VA
Mayo Clinic