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ALN-SOD · GOALS

First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)

Ensayo de REGENERON PHARMACEUTICALS, INC. en Amyotrophic Lateral Sclerosis (ALS) · Mutation in the Superoxide Dismutase-1 (SOD1) Gene (del registro, en inglés).

Fase

Fase 1/2

Estado

Reclutando pacientes

Participantes

54

previstos

Centros

22

Fin del objetivo primario

mar 2032

estimado por el promotor

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, abierto. Comenzó en ago 2024.

Qué mide

Incidence of Treatment-Emergent Adverse Event (TEAEs) in participants treated with ALN-SOD (At week 4 and through week 228) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people. The aim of the study is to see how safe and tolerable the study drug is. The study is looking at several other research questions, including: * The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surrounds the brain and spinal cord, known as Cerebrospinal Fluid (CSF) * How much study drug is in the blood and in the CSF, at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects) * What effects the study drug has on ALS symptoms

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Todos los de REGN, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT06351592), actualizada en sep 2026.