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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

NCT04494256 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 99

Last updated 2025-10-09

Study results available
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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

Conditions

Interventions

DRUG

BIIB105

Administered as specified in the treatment arm.

DRUG

Placebo

Administered as specified in the treatment arm.

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Biogen

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-09-28
Primary Completion
2024-08-13
Completion
2024-08-13
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Italy
  • Netherlands

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04494256 on ClinicalTrials.gov