Ask to take part

Contact the study team using the details below to take part. If there are no contact details below please ask your doctor in the first instance.

Contact Information:

Study Location:

Skip to Main Content
English | Cymraeg
Be Part of Research - Trial Details - TPN-101 in Aicardi-Goutières Syndrome (AGS)

TPN-101 in Aicardi-Goutières Syndrome (AGS)

Not Recruiting

Open to: ALL

Age: 1.0 - N/A

Medical Conditions

Nervous System Malformations
Autoimmune Diseases of the Nervous System
Syndrome


This information is provided directly by researchers, and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information. In some summaries, you may come across links to external websites. These websites will have more information to help you better understand the study.


A phase 2a multi-center, open-label single dose level study of TPN-101 in Patients with Aicardi-Goutières Syndrome (AGS)

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

Mar 2023 Dec 2025

INTERVENTIONAL

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

Intervention Arm Group : Active, TPN-101;



You can take part if:



You may not be able to take part if:


This is in the inclusion criteria above


Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

  • Royal Hospital for Children and Young People
    Edinburgh
    EH9 1LF


The study is sponsored by Transposon Therapeutics, Inc.




We'd like your feedback

Your feedback is important to us. It will help us improve the quality of the study information on this site. Please answer both questions.


Is this study information helpful?

What will you do next?

Read full details for Trial ID: NCT05613868
Last updated 01 April 2025

This page is to help you find out about a research study and if you may be able to take part

You can print or share the study information with your GP/healthcare provider or contact the research team directly.