Ignite Creation Date:
2024-05-06 @ 3:49 PM
Last Modification Date:
2024-10-26 @ 1:57 PM
Study NCT ID:
NCT04768972
Status:
RECRUITING
Last Update Posted:
2024-05-31
First Post:
2021-02-22
Brief Title:
FUSION A Study to Evaluate the Efficacy Safety Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations FUS-ALS
Sponsor:
Ionis Pharmaceuticals Inc
Organization:
Ionis Pharmaceuticals Inc
Study Overview
Official Title:
A Phase 1-3 Study to Evaluate the Efficacy Safety Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients With Fused in Sarcoma Mutations FUS-ALS
Status:
RECRUITING
Status Verified Date:
2024-09
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:
The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis FUS-ALS
Detailed Description:
This is a multi-center three-part study of ION363 in up to 95 participants Part 1 will consist of participants who will be randomized in a 21 ratio to receive a multi-dose regimen of ION363 or placebo for a period of 60 weeks followed by Part 2 in which participants will receive open-label ION363 for a period of 84 weeks Participants may continue to receive open-label ION363 in Part 3 for up to 3 additional years or until ION363 becomes commercially available in the patients country or until the Sponsor discontinues the ION363 development program whichever occurs earlier
Study Oversight
Has Oversight DMC:
None
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?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2020-005522-28 | EUDRACT_NUMBER | None | None |