US Interventional/Drug Trials Only

Updated January 2023

 

ALS TDI updates this list of ALS clinical trials monthly.

 

  Drug Phase Study Design Sponsor
1 Oral edaravone 3
  • no placebo
  • this drug is approved for ALS in the USA and trial is studying a daily dose versus the current 10 days per month dosing
  • recruiting in AZ, AR, CA, CO, FL, GA, IL, LA, MD, MA, MN, NE, NV, NY, PA, TN, TX, VT, VA, WA, WV, WI, WY
 MT Pharma
2 ION363 3
  • 33% chance of placebo for 61 weeks followed by open label extension for
  • antisense oligonucleotide treatment for people carrying a mutation in FUS gene
  • recruiting in CA, MD, MA, MO, NY, OH, UT
 IONIS
3 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
4 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
5 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
6 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
7 Trehalose- Healey Platform Trial Regimen E 2/3
  • 25% chance of placebo for 6 months followed by active treatment on open label extension.
  • drug is delivered intravenously once weekly
  • recruiting in AZ, CA, CO, CT, DC, FL, GA, IL, ID, IN, IA, KS, KY, MD, MA, MI, MN, MO, NE, NY, NC, OH, OR, PA, TN, TX, UT, VA, WA, WI
 Merit Cudkowicz, MD
8 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.
9 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
10 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
11 Fasudil 2
  • no placebo
  • allows participation up to 4 years from symptom onset
  • recruiting in AZ, CO, FL, IL, KY, MO, NY
 Woolsey Pharma
12 L-Serine 2
  • no placebo
  • recruiting in NH
 Elijah W. Stommel MD, PhD
14 Metformin 2
  • no placebo
  • trial is for people that carry a mutation in the C9orf72 gene
  • recruiting in FL
 University of Florida
15 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
16 Prime C 2
  • 33% chance of placebo for 6 months followed by a 12 month open label extension
  • recruiting in CA, MA
 NeuroSense Therapeutics
17 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
18 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
19 Vitamin E, N-acetyl cysteine, L-cystine, nicotinamide, and taurursodiol 2
  • no placebo
  • recruiting in TX
 Dallas VA Medical Center
20 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
21 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
22 ABBV-CLS-7262 1
  • placebo-controlled study for one month followed by 44 weeks of open label treatment
  • recruiting in CA, FL, MA, MN
 Calico Life Sciences
23 baricitinib 1
  • no placebo
  • includes people living with different neurodegenerative diseases including ALS
  • recruiting in MA
Massachusetts General Hospital
24 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
25 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
26 Perampanel pilot
  • 50% chance of placebo
  • trial duration is 9 months
  • recruiting in NY
 Stony Brook University