Safety and blood levels of a new tablet formulation of AUT00206
Research type
Research Study
Full title
A randomised, double-blind, placebo-controlled, single and repeated dose escalation study to assess the safety, tolerability, pharmacokinetics and food effect of a new formulation of AUT00206 in healthy men and women (HMR code: 21-009)
IRAS ID
1003888
Contact name
Aleksandra Gawor
Contact email
Sponsor organisation
Autifony Therapeutics Limited
Eudract number
2021-003211-26
ISRCTN Number
ISRCTN80267019
Research summary
AUT00206 is an experimental treatment for Fragile X syndrome (FXS). FXS is the most common inherited cause of learning disability, and causes a wide range of problems with learning and behaviour. Currently, there are no medicines that specifically treat FXS. AUT00206 acts at sites on nerve cells (called voltage-gated potassium channels) that help to control electrical signalling in parts of the brain involved in learning and behaviour. So, we hope it will be a useful treatment for FXS.
We’re doing this study to test a new tablet form of AUT00206 – a capsule form has already been tested in previous studies. We hope that the new tablet form will be better absorbed into the bloodstream, and give higher blood levels of AUT00206 than the previous capsule form. We aim to find out the side effects and blood levels of AUT00206, and whether food affects blood levels of AUT00206. The tablet form has never been given to humans before, so we’ll start with a small dose, and increase as the study progresses, if previous doses are shown to be safe.
The study is in 3 parts (Parts A1, A2 and B). In Parts A1 and A2, we’ll test single doses of AUT00206 in healthy men and women, aged 18-45. In Part A1, we’ll recruit up to 8 healthy men. Participants will have up to 4 study sessions and take about 11 weeks to finish the study. In Part A2, we’ll recruit up to 8 healthy women. Participants will have up to 2 study sessions and take about 6 weeks to finish the study. In each session, they’ll make 4 outpatient visits and stay on the ward for 3 nights.
In Part B, we’ll test repeated doses of AUT00206 in up to 32 healthy men. Participants will have a single study session and take up to 4 weeks to finish the study. They’ll make 4 outpatient visits and stay on the ward for a minimum of 7 nights and a maximum of 10 nights.
A pharmaceutical company – Autifony Therapeutics Limited, UK is funding the study.
This study will take place at 1 centre in London.
REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
22/YH/0183
Date of REC Opinion
20 Aug 2021
REC opinion
Further Information Favourable Opinion