US Interventional/Drug Trials Only

Updated Deceber 2023


 

ALS TDI updates this list of ALS clinical trials monthly.

 

  Drug Phase Study Design Sponsor
1 ION363 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 (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

ABBV-CLS-7262

Regimen F

Healey Platform Trial

2/3
  • 25% chance of placebo for 6 months followed by active treatment extension for all participants
  • 2 doses are being tested
  • recruiting in AZ, CA, CO, CT, DC, FL, GA, ID, IL, IA, KS, KY, MD, MA, MI, MN, MO, NE, NH, NJ, NY, NC, OH, OR, PA, TN, TX, UT, VA, WA, WI
Merit Cudkowicz, MD
5

DNL 343

Regimen G

Healey Platform Trial

2/3
  • 25% chance of placebo for 6 months followed by active treatment extension for all participants
  • 2 doses are being tested
  • recruiting in AZ, CA, CO, CT, DC, FL, GA, ID, IL, IA, KS, KY, MD, MA, MI, MN, MO, NE, NH, NJ, NY, NC, OH, OR, PA, TN, TX, UT, VA, WA, WI
Merit Cudkowicz, MD

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
MediciNova
7
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
8 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
9 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
10
2
  • 33% chance of placebo, 2 doses being tested
  • 24 weeks trial followed by 24 weeks of open label extension
  • recruiting in NJ
Corcept Therapeutics
11 Metformin 2
  • no placebo
  • trial is for people that carry a mutation in the C9orf72 gene
  • recruiting in FL
University of Florida
12 PTC857 2
  • 50% chance of placebo for 24 weeks followed by 28 weeks of open label extension
  • recruiting in CA, FL, GA, NE, TX, WI
PTC Therapeutics
13 Vitamin E, N-acetyl cysteine, L-cystine, nicotinamide, and taurursodiol 2
  • no placebo
  • recruiting in TX
Dallas VA Medical Center
14 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
15 baricitinib 1
  • no placebo
  • includes people living with different neurodegenerative diseases including ALS
  • recruiting in MA
Massachusetts General Hospital
16
1
  • placebo controlled trial
  • umbilical cord derived T regulatory cells delivered intravenously
  • recruiting in NY
Cellenkos
17  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