US Interventional/Drug Trials Only

Updated February 2023

 

ALS TDI updates this list of ALS clinical trials monthly.

 

  Drug Phase Study Design Sponsor
1 ION363 3
  • 33% chance of placebo for 61 weeks followed by open label extension for 85 weeks
  • 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, IL, KY, MD, MA, NY, PA
 AB Sciences
3 Reldesemtiv 3
  • 33% chance of placebo for 6 months followed by open label extension
  • recruiting in AZ, CA, CO, DC, FL, IL, IN, IA, KS, MD, MA, MI, MO, NE, NY, NC, OH, OR, PA, TN, TX, VT, WI
 Cytokinetics
4 Tofersen (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
5 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, WA
 MediciNova
6 ANX005
 2
  • no placebo
  • drug is delivered intravenously for 22 weeks with additional 14 weeks of followup
  • recruiting in AZ, CA, FL, NY
 Annexon, Inc.
7 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
8 BLZ945
 2
  • no placebo
  • multiple groups or “cohorts” with cohorts 1-4 all receiving treatment and PET imaging, while in cohort 5 participants will be split into two groups that will either receive PET imaging or contribute to spinal fluid analysis
  • recruiting in CT, MA, NY
 Novartis
9 Fasudil
 2
  • no placebo
  • allows participation up to 4 years from symptom onset
  • recruiting in AZ, CO, FL, IL, KY, MO, NY
 Woolsey Pharma
10 Metformin
 2
  • no placebo
  • trial is for people that carry a mutation in the C9orf72 gene
  • recruiting in FL
 University of Florida
11 PTC857
 2
  • 50% chance of placebo for 24 weeks followed by 24 weeks of open label extension
  • recruiting in CA, FL, KS, TX
 PTC Therapeutics
12 SAR443820
 2
  • 33% chance of placebo for 24 weeks followed by 82 weeks of open label treatment
  • recruiting in CA, CO, FL, MA, NY, PA, UT, WI
 Sanofi
13 TPN-101
 2
  • placebo-controlled trial
  • 6 week screening period, followed by 24 weeks of placebo-controlled trial
  • trial is for people that carry a mutation in the C9orf72 gene
  • recruiting in CA, MD, MA, MN, NY, NC
 Transposon Therapeutics
14 Vitamin E, N-acetyl cysteine, L-cystine, nicotinamide, and taurursodiol
 2
  • no placebo
  • recruiting in TX
 Dallas VA Medical Center
15 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
16 Rapa-501
 1/2
  • 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
17 baricitinib
 1
  • no placebo
  • includes people living with different neurodegenerative diseases including ALS
  • recruiting in MA
Massachusetts General Hospital
18 BIIB105
 1
  • placebo controlled trial
  • antisense oligonucleotide treatment for people with sporadic ALS as well as people who carry the ataxin-2 gene mutation
  • recruiting in CA, FL, GA, MD, MA, MO, PA, UT
 Biogen
19 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
20 Perampanel
 pilot
  • 50% chance of placebo
  • trial duration is 9 months
  • recruiting in NY
 Stony Brook University