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Trial in MCT8 deficiency

Egetis have just started active enrolment into the Phase III ReTRIACt study for male participants with MCT8 deficiency and are exploring options to refer any participants from a Dutch speaking background from Belgium to the PI (Dr Visser) at the active EMC site in Rotterdam, Netherlands.

The ReTRIACt trial (clinicaltrials.gov identifier NCT05579327 Study Record | Beta ClinicalTrials.gov) is a double-blind, randomized Phase 3 multicenter placebo-controlled study in at least 16 evaluable male participants diagnosed with MCT8 deficiency. 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, or if they are tiratricol treatment-naïve. Participants, from 4 years of age and having demonstrated stable maintenance treatment with Emcitate, will be randomized to receive placebo or Emcitate 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 that, for participants in the placebo group, removal of Emcitate will lead to an increase of serum total T3 concentration above the ULN and requirement of rescue treatment with Emcitate, compared to those who continue to receive tiratricol.

The study includes mostly home nurse visits, so it would be anticipated that only about 4 clinic visits to the EMC site in Rotterdam would be needed and all other visits will be conducted at home/at the place of the participants’ residence.

If you are aware of any potential participants that might meet these criteria and with physicians supportive of such a referral, we would be very interested to follow-up on these opportunities.

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