|
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 |