Ignite Creation Date:
2025-12-24 @ 11:28 PM
Ignite Modification Date:
2025-12-25 @ 9:15 PM
Study NCT ID:
NCT04494256
Status:
TERMINATED
Last Update Posted:
2025-10-09
First Post:
2020-07-30
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
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
Sponsor:
Biogen
Organization:
Study Overview
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:
TERMINATED
Status Verified Date:
2025-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Sponsor's decision
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ALSpire
Brief 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
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).
\- 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).
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2020-000207-36 | EUDRACT_NUMBER | None | View |