NCT04494256

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

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: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 99 (actual)
• Sponsor: Biogen · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Detailed 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).

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	BIIB105	Administered as specified in the treatment arm.
DRUG	Placebo	Administered as specified in the treatment arm.

Timeline

• Start date: 2020-09-28
• Primary completion: 2024-08-13
• Completion: 2024-08-13
• First posted: 2020-07-31
• Last updated: 2025-10-09
• Results posted: 2025-10-09

Locations

17 sites across 4 countries: United States, Canada, Italy, Netherlands

Regulatory

• FDA-regulated drug study