Ignite Creation Date:
2025-12-24 @ 4:31 PM
Ignite Modification Date:
2026-01-03 @ 9:40 PM
Study NCT ID:
NCT06153966
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-12-27
First Post:
2023-11-21
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Sponsor:
Ionis Pharmaceuticals, Inc.
Organization:
Study Overview
Official Title:
A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-12
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 purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
Detailed Description:
This is a first-in-human, randomized, multi-center study in participants with prion disease. Eligible participants will receive ION717 and placebo. The study will consist of a screening period of up to 6 weeks, a 30-week double-blind treatment period, a 70-week open-label extension period and a 32-week post-treatment period. During the double-blind period, the order of ION717 and placebo doses will be randomized and blinded to participants, study sites and the Sponsor. During the open-label extension period, all participants will receive ION717. Multiple dose levels will be tested.
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 |
|---|---|---|---|---|
| 2023-503355-98 | EUDRACT_NUMBER | None | View |