US Interventional Trials Only

Updated April 29, 2025

 

ALS TDI updates this list of ALS clinical trials monthly.

 

  Drug Phase Study Design Sponsor
1 ION363 (ulefnersen) 3
  • antisense oligonucleotide therapy for genetic FUS ALS
  • sites in CA, MD, MA, MO, NY, OH, UT
Ionis Pharmaceuticals, Inc.
2 MN-166 (ibudilast) 2/3
  • 50% chance of placebo for 12 months followed by an open label extension for 6 months
  • sites in CA, FL, GA, IN, MD, MN, NY, NC, PA, VA
MediciNova
3 RAPA-501 2/3
  • no placebo
  • autologous hybrid TREG/Th2 cells delivered intravenously
  • Expanded Access Program also available for those ineligible for this trial
  • site in MA
Rapa Therapeutics LLC
4 VHB937 2
  • 33% chance of placebo for 40 weeks treatment followed by an open label extension
  • intravenous delivery of an antibody designed to activatea protein called TREM2
  • site in TX
Novartis Pharmaceuticals
5 ranolazine
2
  • placebo controlled trial evaluating two doses of ranolazine
  • site in FL, KS, MO
Swathy Chandrashekhar, MD
6 AMT-162 1/2
  • no placebo
  • gene therapy for SOD1 relatedALS
  • one-time intrathecal injection, with up to five years of follow-up
  • participants cannot be on tofersen at trial initiation
  • sites in CA, MA, NY sur
UniQure Biopharma B.V.
7 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
  • site in FL
University of Florida
8 CNS10-NPC-GDNF 1
  • no placebo
  • cells that produce GDNF (a growth factor) will be surgically
  • transplanted into motor cortex of brain
  • site in CA
Cedars-Sinai Medical Center
9
1
  • no placebo
  • site in MD
Johns Hopkins University
10
prosetin 1
  • placebo controlled trial lasting 14 days followed by open label extension
  • oral drug designed to target MAP4 kinase
  • site in MA
ProJenX
11
N/A
  • no placebo
  • trial is evaluating multi-site direct current stimulation
  • site in MA
PathMaker Neurosystems Inc.
12 Spinal Cord Associative Plasticity (SCAP)
N/A
  • trial being conducted at Bronx Veterans Administration Medical Center
  • 2-4 month run in period to optimize SCAP, followed by a 2 week period of SCAP with or without hand exercises
  • site in NY
VA Office of Research and Development
13 RAPA-501 EAP
  • Expanded Access Program for people that are not eligible for trials
  • autologous hybrid TREG/Th2 cells delivered intravenously
  • sites in MA, NJ
Rapa Therapeutics LLC
14 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 FL
Mayo Clinic