Ignite Creation Date:
2025-12-24 @ 5:53 PM
Ignite Modification Date:
2025-12-29 @ 1:03 PM
Study NCT ID:
NCT05613868
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-04-04
First Post:
2022-11-07
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
TPN-101 in Aicardi-Goutières Syndrome (AGS)
Sponsor:
Transposon Therapeutics, Inc.
Organization:
Study Overview
Official Title:
A Phase 2a Study of TPN-101 in Patients With Aicardi-Goutières Syndrome (AGS)
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-04
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:
A phase 2a multi-center, open-label single dose level study of TPN-101 in Patients with Aicardi-Goutières Syndrome (AGS)
Detailed Description:
The study is planned in pediatric and adult patients with AGS that are greater than 1 year and weigh at least 10 kg. The TPN-101 dose will be adjusted from 100 mg to 400 mg based on weight to achieve similar drug exposures in all subjects. The study plans to enroll 10 - 16 subjects. This study includes a 6-8 week Screening Period, a 48-week Open label Treatment Period, and a 12-week Follow-up Period.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: