Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial.

Publication

Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial.

Journal Paper/Review - May 20, 2024

Benatar Michael, Hansen Thomas, Rom Dror, Geist Marie A, Blaettler Thomas, Camu William, Kuzma-Kozakiewicz Magdalena, van den Berg Leonard H, Juntas-Morales Raul, Chio Adriano, Andersen Peter Munch, Pradat Pierre-Francois, Lange Dale J, Van Damme Philip, Mora Gabriele, Grudniak Mariusz, Elliott Matthew, Petri Susanne, Olney Nicholas, Ladha Shafeeq S, Goyal Namita A, Meyer Thomas, Hanna Michael G, Quinn Colin, Genge Angela, Zinman Lorne, Jabari Duaa, Shoesmith Christen, Ludolph Albert C., Neuwirth Christoph, Nations Sharon, Shefner Jeremy M, Turner Martin R, Wuu Joanne, Bennett Richard, Dang Hoang, Sundgreen Claus, ORARIALS-01 trial team

Citation

Benatar M, Hansen T, Rom D, Geist M, Blaettler T, Camu W, Kuzma-Kozakiewicz M, van den Berg L, Juntas-Morales R, Chio A, Andersen P, Pradat P, Lange D, Van Damme P, Mora G, Grudniak M, Elliott M, Petri S, Olney N, Ladha S, Goyal N, Meyer T, Hanna M, Quinn C, Genge A, Zinman L, Jabari D, Shoesmith C, Ludolph A, Neuwirth C, Nations S, Shefner J, Turner M, Wuu J, Bennett R, Dang H, Sundgreen C, ORARIALS-01 trial team. Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial. Lancet Neurol 2024; 23:687-699.

Type

Journal Paper/Review (English)

Journal

Lancet Neurol 2024; 23

Publication Date

May 20, 2024

Issn Electronic

1474-4465

Pages

687-699

Brief description/objective

Amyotrophic lateral sclerosis is a progressive neurodegenerative disorder leading to muscle weakness and respiratory failure. Arimoclomol, a heat-shock protein-70 (HSP70) co-inducer, is neuroprotective in animal models of amyotrophic lateral sclerosis, with multiple mechanisms of action, including clearance of protein aggregates, a pathological hallmark of sporadic and familial amyotrophic lateral sclerosis. We aimed to evaluate the safety and efficacy of arimoclomol in patients with amyotrophic lateral sclerosis.