A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation — ALSpire  

Amyotrophic Lateral Sclerosis Clinical Trial

Official title: A Phase 1/2 Multiple-Ascending-Dose Study With a Long-Term Open-Label Extension to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effect on Disease Progression of BIIB105 Administered Intrathecally to Adults With Amyotrophic Lateral Sclerosis With or Without Poly-CAG Expansion in the ATXN2 Gene

Status Active, not recruiting

Clinical Trial Summary

The ALSpire Study is a clinical trial evaluating the investigational drug BIIB105 in adults living with amyotrophic lateral sclerosis (ALS).

The ALSpire Study consists of two parts:

• Part 1: 6-month placebo-controlled study. During Part 1, participants are randomly assigned to receive either BIIB105 or placebo in a 3:1 or 2:1 ratio (depending on the participant's assigned Cohort).

• Part 2: up to 3-year long-term open-label extension. During Part 2, all participants receive BIIB105.

The objectives of the study are to evaluate:

• The safety and tolerability of BIIB105 in people with ALS

• What the body does to BIIB105 (also called "pharmacokinetics")

• What BIIB105 does to the body (also called "pharmacodynamics")

• Whether BIIB105 can slow the worsening of clinical function

Clinical Trial Description

About BIIB105:

• BIIB105 is an investigational drug designed to reduce the levels of a protein called ATXN2. It is administered intrathecally (via a procedure called lumbar puncture). ;

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Disease Progression
• Motor Neuron Disease
• Sclerosis

• NCT number: NCT04494256
• Study type: Interventional
• Source: Biogen
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: September 28, 2020
• Completion date: July 15, 2026