US Interventional/Drug Trials Only

Updated January 30, 2026

 

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 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.
3 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
4 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, CT, VA
 Zydus Therapeutics Inc.
5 Vit E, N-Ac cysteine, L-cystine, Nicotinamide, Taurursodiol 2
  • no placebo
  • site in TX
 Dallas VA Medical Center
6 ranolazine 2
  • placebo controlled trial evaluating two doses of ranolazine
  • sites in CA, FL, KS, OH, MO
 Swathy Chandrashekhar, MBBS
7 Tofersen 2
  • phase 2 study testing tofersen (Qalsody) in non-SOD1 and non-FUS related cases of sporadic and familial ALS
  • must have done genetic testing to confirm absence of SOD1 and FUS mutations
  • site in MO
 Washington University School of Medicine
8 MTP-101C 1/2
  • no placebo
  • site in NC
 Duke University
9 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
10 VTx-002 1/2
  • Vectorized antibody treatment targeting TDP-43 pathology
  • Two escalating dose (low dose and high dose) cohorts are planned 
  • The duration of the study will be a maximum of 5 years and 5 weeks (265 weeks) for each participant
  • site in MA
 Vector Y Therapeutics
11 prosetin 1
  • placebo controlled trial lasting 14 days followed by open label extension
  • oral drug designed to target MAP4 kinase
  • site in MA
 ProJenX
12 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.
13 acamprosate 1
  • no placebo
  • recruiting people with C9orf72-related ALS 
  • site in MD
 National Institute of Neurological Disorders and Stroke (NINDS)
14 RAPA-501
  • 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
15 SPG302
  • Expanded Access Program for people that are not eligible for trials
  • Once a day oral pill that aims to restore synapses
  • Sites in AL, AZ, CA, FL, IL, MA, NH, NJ, NY, PA, VA
 Spinogenix
16 Psilocybin Early 1
  • no placebo
  • site in MD
 Johns Hopkins University
17 MN-166 (ibudilast)
  • 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
18 terazosin Early 1
  • no placebo
  • single center, open label pilot study to assess the safety and tolerability of terazosin (TZ) at a dose of 5 milligrams (mg) per os (PO) daily for patients with ALS
  • site in IA
 University of Iowa