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ReTRIACt trial design presented at the Annual Meeting of the European Society for Paediatric Endocrinology

September 25, 2023

Stockholm, Sweden, September 25, 2023. Egetis Therapeutics AB (publ) (Nasdaq Stockholm: EGTX) announces the poster presentation of the ReTRIACt trial design at the 61st Annual Meeting of the European Society for Paediatric Endocrinology (ESPE 2023), which was held in The Hague, The Netherlands, September 21-23, 2023. At home care and patient needs were at the core of the design to provide optimal comfort and retention for the participating families. The ReTRIACt trial aims to study the effects of tiratricol treatment withdrawal in MCT8 deficiency which is an ultra-rare condition that causes severe disabilities. This trial was requested by the US FDA as pivotal for the New Drug Application submission for tiratricol.
The patient-centred design of the ReTRIACt trial has taken into careful consideration the challenges of enrolling patients from small populations who cannot travel or mobilize independently. The trial uses specialist domiciliary nurses and home-based monitoring technology so that it can be conducted primarily in patients’ homes. The first patients were enrolled in the trial in the third quarter of 2023 and the trial is estimated to complete patient enrolment during 2023. Topline results are expected during the first half of 2024.

Nicklas Westerholm, CEO of Egetis, commented: “Studying rare diseases is a challenge due to the smaller patient populations. For the vulnerable MCT8 deficient patient population there are further challenges posed by the disabilities that the disorder causes. It was a priority for us to ensure that the ReTRIACt trial was truly accessible. We developed the trial with the patients and their carers in mind, in collaboration with a patient association. This led to its home-based approach which should allow more patients to enrol and stay in the trial, while avoiding unnecessary disruption to their quality of life. The data collated will support our US FDA New Drug Application submission for tiratricol for this patient population.
Poster Title:
Effects of tiratricol treatment withdrawal in monocarboxylate transporter 8 (MCT8) deficiency: ReTRIACt trial
M. Freund, K. Chatterjee, F. van der Most, D. Bowers, A. Persson, W. E. Visser, A. J. Bauer
The poster can be accessed here.
About the ReTRIACt trial
The ReTRIACt trial ( identifier NCT05579327) is a double-blind, randomized Phase 3 multicenter placebo-controlled study in at least 16 evaluable male participants diagnosed with MCT8 deficiency. This trial was requested by the US FDA as pivotal for the New Drug Application submission for tiratricol. The study protocol starts with an open-label treatment period in which a stable maintenance dose of tiratricol, essential for progression into the Randomized Treatment Period, will be established. The duration of the initial open-label treatment period will vary depending on whether the participant is currently receiving treatment with tiratricol at the time of enrollment in the study (Cohort A), or if they are considered to be tiratricol treatment-naïve (Cohort B). Participants are considered to be tiratricol-naïve if they have never previously been administered tiratricol or have previously received tiratricol but are not receiving tiratricol at the time of enrollment. Participants, from four years of age and having demonstrated stable maintenance treatment with tiratricol, will be randomized to receive placebo or tiratricol for 30 days or until reaching rescue criterion (serum total triiodothyronine [T3] above upper limit of normal [ULN] of the participant's normal range, for a sample collected during the 30-day Randomized Treatment Period). The research hypothesis to be tested is as follows: for participants in the placebo group, removal of tiratricol will lead to an increase of serum total T3 concentration above the ULN and requirement of rescue treatment with tiratricol, compared to those who continue to receive tiratricol.
For more information about MCT8 deficiency, please see



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