US Interventional/Drug Trials Only

Updated September 11, 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 AZ, CA, CO, FL, GA, IL, KS, MD, MA, MO, NE, NY, PA, TX, WA

 Prilenia
2 COYA 302 2/3
  • 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 AZ, CA, FL, GA, IL, MD, MA, MI, MO, NE, NY, PA, TX
Coya Therapeutics
3 RAPA-501 2/3
  • no placebo
  • autologous hybrid TREG/Th2 cells delivered intravenously
  • site in MA
 Rapa Therapeutics LLC
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 CA, CT, FL, GA, MA, MI, NE, NC, PA, TX, VA, WA
 Zydus Therapeutics Inc.
5 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, AZ
 PhenoNet, Inc.
6 ranolazine 2
  • placebo controlled trial evaluating two doses of ranolazine
  • sites in CA, FL, KS, OH, MO
 Swathy Chandrashekhar, MBBS
7 CORT113176 (dazucorilant) 2
  • 6 month placebo-controlled study followed by a six month open label extension
  • less than 50% placebo
  • sites in AZ, CA, NJ, NY
 Corcept Therapeutics
8 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
  • sites in MO, GA, MA
 Washington University School of Medicine
9 MTP-101C 1/2
  • no placebo
  • site in NC
 Duke University
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
  • sites in CA, FL, MA, PA
 Vector Y Therapeutics
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 INS1202 1
  • no placebo
  • recruiting SOD1 and Sporadic patients
  • sites in MO, CA, OH, PA, MA, MD
 Insmed Gene Therapy LLC
13 Psilocybin Early 1
  • no placebo
  • site in MD
 Johns Hopkins University
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 AL, AZ, CA, FL, IL, NH, NJ, NM, NY, PA, TX, VA
 Spinogenix
15 CNM-Au8 EAP
  • Expanded Access Program for people that are not eligible for trials
  • Oral liquid gold nanoparticle suspension, aimed at enhancing energy production in neurons and oligodendrocyte
  • Sites in NC
 Clene Nanomedicine
16 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