Ignite Creation Date:
2024-05-06 @ 7:50 PM
Last Modification Date:
2024-10-26 @ 3:14 PM
Study NCT ID:
NCT06150716
Status:
RECRUITING
Last Update Posted:
2024-06-21
First Post:
2023-11-21
Brief Title:
Orbit Study A Study to Evaluate the Safety Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease PMD
Sponsor:
Ionis Pharmaceuticals Inc
Organization:
Ionis Pharmaceuticals Inc
Study Overview
Official Title:
A Phase 1b Study to Evaluate the Safety Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease
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 safety and tolerability of ION356
Detailed Description:
This is a Phase 1b open-label multiple-ascending dose MAD study of ION356 in approximately 24 pediatric participants with Pelizaeus-Merzbacher Disease and genetic confirmation of proteolipid protein 1 PLP1 gene duplication The study will have 2 parts a 48-week multiple-ascending dose MAD part followed by an open-label long-term extension LTE part of 109 weeks Eligible participants during the MAD portion of the study will receive doses of ION356 and upon completion will seamlessly transition to the open-label LTE to receive doses of ION356
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?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2022-502432-39 | EUDRACT_NUMBER | None | None |