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
Public ClinicalTrials.gov record NCT04494256. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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
Study identification
- NCT ID
- NCT04494256
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 99 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 28, 2020 (Actual)
- Primary completion
- Aug 13, 2024 (Actual)
- Completion
- Aug 13, 2024 (Actual)
- Last update posted
- Oct 9, 2025
2020 – 2024
United States locations
- U.S. sites
- 14
- U.S. states
- 12
- U.S. cities
- 14
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Barrow Neurological Institute | Phoenix | Arizona | 85013 | — |
| University of California San Diego Medical Center | La Jolla | California | 92037 | — |
| Stanford Neuromuscular Research Center | Palo Alto | California | 94305 | — |
| University of Colorado Hospital - Neuroscience Center -Anschutz Medical Campus | Aurora | Colorado | 80045 | — |
| Georgetown University | Washington D.C. | District of Columbia | 20007-2113 | — |
| Mayo Clinic Florida | Jacksonville | Florida | 32224 | — |
| Orlando Health | Orlando | Florida | 32806 | — |
| The Emory Clinic | Atlanta | Georgia | 30322 | — |
| ALS Clinic - Department of Neurology, Neuromuscular Division, Johns Hopkins University, School of Medicine | Baltimore | Maryland | 21218 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| Washington University, School of Medicine | St Louis | Missouri | 63110 | — |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
| Houston Methodist Neurological Institute | Houston | Texas | 77030 | — |
| University of Utah | Salt Lake City | Utah | 84132 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04494256, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 9, 2025 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04494256 live on ClinicalTrials.gov.