Treatment trials
There are many clinical trials investigating various treatments for MND across the world. Here we focus on trials that are in the spotlight and provide you with updates on the latest results and related news.
Unsure of some of the wording used in these clinical trials pages? Our glossary gives easy to understand definitions of commonly used words when discussing clinical trials. You can access the clinical trial glossary.
United Kingdom Trials
AMX0035
Evaluating TUDCA (tauroursodeoxycholic acid) and sodium phenylbutyrate (PB)
Phase 3 (PHOENIX)
Recruiting status: Open
BIIB067 (Tofersen)
Evaluating Tofersen to treat familial MND caused by mistakes in the SOD1 gene
Phase 3 (ATLAS)
Recruiting status: Open
ILB
Evaluating ILB
Phase 2
Recruiting status: Closed
Interleukin-2
Evaluating Interleukin-2
Phase 2 (MIROCALS)
Recruiting status: Closed
Ion363/Jacifusen
Evaluating ION363 to treat MND caused by mistakes in the FUS gene
Phase 3 (FUS-ALS)
Recruiting status: Open
MAGNET Platform Trial
Evaluating multiple treatments simultaneously
Lithium Carbonate (PRELUDE)
Recruiting status: In preparation
MND SMART
Evaluating multiple treatments simultaneously
Phase 2 - Memantine Hydrochloride
Phase 2 - Trazodone Hydrochloride
Recruitment status: Open
Oral Edaravone
Evaluating the formulation FAB122 of oral edaravone
Phase 3 (ADORE)
Recruiting status: Open
Pegcetacoplan/APL-2
Evaluating pegcetacoplan/APL-2
Phase 2 (MERIDIAN)
Recruiting status: Closed
Reldesemtiv/Tirasemtiv
Evaluating Reldesemtiv/Tirasemtiv
Phase 3 (COURAGE-ALS)
Recruiting status: Open
SAR443820
Evaluating SAR443820
Phase 2 (HIMALAYA)
Recruiting status: In preparation
Tauroursodeoxycholic acid
Evaluating tauroursodeoxycholic acid (TUDCA)
Phase 3 (TUDCA-ALS)
Recruitment status: Closed
Trimetazidine
Evaluating Trimetazidine Dihydrochloride
Phase 2 (MetFlex)
Recruiting status: In preparation
Triumeq
Evaluating Triumeq
Phase 3 (LIGHTHOUSE 2)
Recruiting status: Open
WVE-004
Evaluating WVE-004 to treat familial MND caused by mistakes in the C9orf72 gene
Phase 1/2 (FOCUS-C9)
Recruiting status: Open
International Trials
CNM-Au8
Evaluating CNM-Au8 (gold nanocrystals)
Phase 2 (RESCUE-ALS)
Recruitment status: Closed
CuATSM
Evaluating CuATSM
Phase 2/3
Recruiting status: Closed
Edaravone/Radicut/Radicava
Evaluating Edaravone
Phase 3
Recruiting status: Open
Healey Platform Trial
Evaluating multiple treatments simultaneously
Recruitment status: Open
Ibuldilast/MN-166
Evaluating Ibudilast
Phase 2b/3
Recruiting status: Open
Masitinib
Evaluating Masitinib
Phase 3
Recruiting status: Open
Memantine
Evaluating Memantine
Phase 3
Recruiting status: Closed
Metformin
Evaluating Meformin to treat familial MND caused by mistakes in the C9orf72 gene
Phase 2
Recruiting status: Open
RT1001
Evaluating RT1011 (modified linoleic acid)
Phase 2
Recruiting status: Open
Completed Trials
Arimoclomol
Evaluating Arimoclomol
Phase 3
Levosimendan (ODM-109)
Evaluating Levosimendan
Phase 3 (REFALS)
NP001
Evaluating NP001
Phase 2
NurOwn
Evaluating NurOwn
Phase 3
Nuedexta
Evaluating Nuedexta
Phase 3
Ravulizumad/Ultomiris
Evaluating Ravulizumab/Ultomiris
Phase 3
Due to the sheer number of clinical trials investigating treatments for MND taking place at the moment, we have not included every trial. Please use the button below to find out about more MND trials currently recruiting around the world.
This link will take you to clinicaltrials.gov, the largest clinical trials database.
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Last updated: 06/09/2022