NCT05613868

TPN-101 in Aicardi-Goutières Syndrome (AGS)

A Phase 2a Study of TPN-101 in Patients With Aicardi-Goutières Syndrome (AGS)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Transposon Therapeutics, Inc. · Industry
• Sex: All
• Age: 12 Months
• Healthy volunteers: Not accepted

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.

Conditions

• Aicardi-Goutières Syndrome (AGS)

Interventions

Type	Name	Description
DRUG	TPN-101	100 mg/ day up to 400mg/day of TPN-101

Timeline

• Start date: 2023-03-15
• Primary completion: 2025-02-27
• Completion: 2025-02-27
• First posted: 2022-11-14
• Last updated: 2026-04-08

Locations

5 sites across 3 countries: France, Italy, United Kingdom