USA Interventional Trials

Updated June 10, 2025



  Drug Phase Study Design Sponsor
1 ION363 (ulefnersen) 3
  • antisense oligonucleotide treatment for people carrying a mutation in FUS gene
  • 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
  • site in MA
Rapa Therapeutics LLC
4 ranolazine 2
  • placebo controlled trial evaluating two doses of ranolazine
  • sites in CA, FL, KS, MO
Swathy Chandrashekhar, MD
5 VHB937 2
  • 33% chance of placebo for 40 weeks followed by an open label extension
  • intravenous delivery of an antibody designed to activate TREM2 protein
  • site in TX
Novartis Pharmaceuticals
6 AMT-162 1/2
  • no placebo
  • one-time gene therapy for SOD1-related ALS
  • up to five years of follow-up
  • sites in CA, MA, NY
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
81
  • 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 DC, MA, TN 
Amylyx Pharmaceuticals Inc.
9
1
  • no placebo
  • cells that produce GDNF (a neurotrophic factor) are surgically transplanted into motor cortex of brain
  • site in CA
Cedars-Sinai Medical Center
10 prosetin 1
  • placebo controlled trial lasting 14 days followed by long term open label extension
  • oral drug designed to target MAP4 kinase
  • site in MA
ProJenX
11
1
  • no placebo
  • site in MD
Johns Hopkins University
12
N/A
  • no placebo
  • trial evaluates multi-site direct current stimulation
  • site in MA
PathMaker Neurosystems Inc.
13
N/A
  • 2-4 week run in period followed by 2 weeks of SCAP with or without hand exercises
  • trial being conducted at Bronx Veterans Administration Medical Center
  • site in NY
VA Office of Research and Development
14 RAPA-501 EAP
  • 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 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 information here
Clene Nanomedicine
16
EAP
  • Expanded Access Program
  • Contact Spinogenix for more information here
Spinogenix, Inc.