Ignite Creation Date:
2025-12-24 @ 3:32 PM
Ignite Modification Date:
2025-12-31 @ 7:55 PM
Study NCT ID:
NCT06351592
Status:
RECRUITING
Last Update Posted:
2025-09-17
First Post:
2024-04-02
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
Sponsor:
Regeneron Pharmaceuticals
Organization:
Study Overview
Official Title:
First in Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ALN-SOD in Participants With Amyotrophic Lateral Sclerosis and SOD1 Mutations
Status:
RECRUITING
Status Verified Date:
2025-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with amyotrophic lateral sclerosis (ALS) caused by a change in a gene called the superoxide dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people. The aim of the study is to see how safe and tolerable the study drug is.
The study is looking at several other research questions, including:
* The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surrounds the brain and spinal cord, known as cerebrospinal fluid (CSF)
* How much study drug is in the blood and in the CSF, at different times
* Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
* What effects the study drug has on ALS symptoms
The study is looking at several other research questions, including:
* The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surrounds the brain and spinal cord, known as cerebrospinal fluid (CSF)
* How much study drug is in the blood and in the CSF, at different times
* Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
* What effects the study drug has on ALS symptoms
Detailed Description:
None
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?:
True
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2023-510344-20-00 | REGISTRY | EU CT Number | View |