|
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 |