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

Amyotrophic Lateral Sclerosis (ALS) Clinical Trial

Official title: First in Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ALN-SOD in Participants With Amyotrophic Lateral Sclerosis and SOD1 Mutations

Status Not yet recruiting

Clinical Trial Summary

This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with amyotrophic lateral sclerosis (ALS) who have a mutation 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 molecules 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

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Amyotrophic Lateral Sclerosis (ALS)
• Motor Neuron Disease
• Sclerosis

• NCT number: NCT06351592
• Study type: Interventional
• Source: Regeneron Pharmaceuticals
• Contact: Clinical Trials Administrator
• Phone: 844-734-6643
• Email: clinicaltrials@regeneron.com
• Status: Not yet recruiting
• Phase: Phase 1
• Start date: June 28, 2024
• Completion date: April 28, 2029