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

Tegoprubart (AT-1501)
Evaluating AT-1501 antibody
Phase 2
Recruiting status: Closed

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.

Last updated: 05/10/2022

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