US Interventional/Drug Trials Only

Updated May 2, 2024

 

ALS TDI updates this list of ALS clinical trials monthly.

 

  Drug Phase Study Design Sponsor
1 ION363 (ulefnersen) 3
  • antisense oligonucleotide treatment for people carrying a mutation in FUS gene
  • recruiting in CA, MD, MA, MO, NY, OH, UT
IONIS
2 Masitinib 3
  • 33% chance of placebo
  • trial volunteers will be separated into 3 groups, a high dose with riluzole, a low dose with riluzole, or placebo with riluzole
  • recruiting in AL, CA, KY, MD, MA, VA
AB Sciences
3 Tofersen - ATLAS (for presymptomatic SOD1) 3
  • biomarkers will be monitored over time and tofersen or placebo will be administered if specific biomarker changes occur
  • antisense oligonucleotide treatment for people carrying specific SOD1 mutations, but no current symptoms of ALS
  • recruiting in AZ, CA, FL, GA, IL, MD, MA, MO, NY, TX
Biogen
4 Ibudilast 2/3
  • 50% chance of placebo for 12 months followed by an open label extension for 6 months
  • recruiting in CA, FL, GA, IN, KS, MD, MN, NY, NC, OR, PA, VA
MediciNova
5
Rapa-5012/3
  • no placebo
  • autologous T cells are administered intravenously for 6 months followed by an additional 6 months of observation
  • recruiting in MA, NJ
Rapa Therapeutics
6 AP-101 2
  • placebo-controlled trial
  • drug is delivered intravenously
  • trial is for people carrying SOD1 mutation or sporadic ALS
  • recruiting in CA
AL-S Pharma
7 Vitamin E, N-acetyl cysteine, L-cystine, nicotinamide, and taurursodiol 2
  • no placebo
  • recruiting in TX
Dallas VA Medical Center
8 Istradefylline and AIH 1/2
  • crossover study: all trial volunteers will receive placebo and all trial volunteers will receive active treatment at different timepoints
  • recruiting in FL
University of Florida
9 baricitinib 1
  • no placebo
  • includes people living with different neurodegenerative diseases including ALS
  • recruiting in MA
Massachusetts General Hospital
10
1
  • placebo controlled trial
  • umbilical cord derived T regulatory cells delivered intravenously
  • recruiting in NY, TX
Cellenkos
11 CNS10-NPC-GDNF 1
  • no placebo
  • cells that produce GDNF (a growth factor) will be transplanted into motor cortex of brain
  • recruiting in CA
Cedars-Sinai Medical Center
12CNM-Au8EAP
  • Expanded Access Program for people that are not eligible for trials
  • CNM-Au8 will be administered for up to  96 weeks orally or by feeding tube
  • sites in AZ, CA, CT, FL, IL, KS, MI, NE, NC, OR, PA, TX, 
Clene Nanomedicine
13Rapa 501EAP
  • Expanded Access Program for people that are not eligible for trials
  • Autologous hybrid TREG/Th2 cells delivered intravenously
  • sites in MA, NJ
Rapa Therapeutics

14PridopidineEAP
  • Expanded Access Program for people that are not eligible for trials
  • pridopidine will be administered twice daily by mouth or feeding tube for 2 years
  • sites not yet listed, but contact information is available on clinicaltrials.gov
Prilenia