US Interventional/Drug Trials Only

Updated October 20, 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 RAPA-501 2/3
  • no placebo
  • autologous hybrid TREG/Th2 cells delivered intravenously
  • site in MA
 Rapa Therapeutics LLC
3 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
4 foralumab 2
  • The goal of this clinical trial is to learn whether a nasal spray medicine called foralumab is safe and easy to tolerate in adults with amyotrophic lateral sclerosis (ALS)
  • 12-week randomized, controlled treatment period followed by a 12-week active extension treatment period
  • 3:1 active drug : placebo randomization ratio
  • Sites in MA
 Tiziana Life Sciences LTD
5 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.
6 ranolazine 2
  • placebo controlled trial evaluating two doses of ranolazine
  • sites in CA, FL, KS, OH, MO
 Swathy Chandrashekhar, MBBS
7 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
8 MTP-101C 1/2
  • no placebo
  • site in NC
 Duke University
9 BIIB147 1/2
  • Study to evaluate the safety and tolerability of BIIB147, an investigational antisense oligonucleotide targeting stathmin 2 pre-mRNA in people with broad ALS
  • Recruiting people with sporadic and familial ALS, but SOD1 and FUS are exluded
  • Sites in MO, TX
 Biogen
10 INS1202 1
  • no placebo
  • recruiting SOD1 and Sporadic patients
  • sites in CA, MD, MA, MO, OH, PA
 Insmed Gene Therapy LLC
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 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
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, KS, 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 Psilocybin Early 1
  • no placebo
  • site in MD
 Johns Hopkins University