US Interventional/Drug Trials Only

Updated March 13, 2026

 

ALS TDI updates this list of ALS clinical trials monthly.

 

  Drug Phase Study Design Sponsor
1 Pridopidine 3
  • Randomized (3:2), double-blind, placebo-controlled study followed by an open-label extension period
  • Able to swallow a capsule.
  • Sites in MA, NE, TX

Prilenia
2 RAPA-501 2/3
  • no placebo
  • autologous hybrid TREG/Th2 cells delivered intravenously
  • site in MA
Rapa Therapeutics LLC
3 NUZ-001 2/3
  • Exclusion if family history of SOD1 or VCP-associated ALS
  • Matching placebo
  • Site in MA
Merit E. Cudkowicz, MD
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 CORT113176 (dazucorilant) 2
  • open-label extension (OLE) study
  • less than 50% placebo
  • trial sites across the globe
Corcept Therapeutics
9 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
10 MTP-101C 1/2
  • no placebo
  • site in NC
Duke University
11 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
12 prosetin 1
  • placebo controlled trial lasting 14 days followed by open label extension
  • oral drug designed to target MAP4 kinase
  • site in MA
ProJenX
13 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.
14 INS1202 1
  • no placebo
  • recruiting SOD1 and Sporadic patients
  • site in MO
Insmed Gene Therapy 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