Evicenter
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Emcitate (tiratricol)

MCT8 deficiency

Last updated
78 sources

Product information and disease descriptionPlace of product in therapyApproaches to treatment

Expected outcomes of therapy

3 evidence topics · 2 sources

Thyroid hormone and cardiovascular outcomes across the two studies supporting approval

“The effectiveness of Emcitate was evaluated in two clinical studies in patients ranging in age from infants to adults, including an international, multi-center, randomized, placebo-controlled trial (NCT05579327) and a longer-term open-label study. Across both studies, patients treated with Emcitate had reductions in excess thyroid hormone levels in the bloodstream and improvements in cardiovascular and metabolic symptoms impacted by thyroid levels, such as systolic blood pressure and heart rate.” (opens the source at this quote in a new tab)

Biochemical response: Triac Trial II

“Among key secondary endpoints, total serum thyroid hormone T3 concentrations were reduced significantly and durably in all patients, thereby verifying tiratricol’s ability to alleviate thyrotoxicosis in MCT8 deficiency patients.” (opens the source at this quote in a new tab)

Neurodevelopmental outcomes: Triac Trial II

“The numerical improvements versus baseline observed on the primary endpoints of neurocognitive development assessed by the GMFM-88 and BSID-III scales did not show a statistically significant improvement versus historical controls.” (opens the source at this quote in a new tab)

“The co-primary endpoints were assessed by changes in the Gross Motor Function Measure (GMFM)-88 total score and the Bayley Scales for Infant and toddler Development (BSID)-III Gross Motor Skill domain, compared to natural history scores from the Triac Trial I.” (opens the source at this quote in a new tab)

“Patients were initially treated for 96 weeks with tiratricol, and the treatment effect was evaluated after 96 weeks.” (opens the source at this quote in a new tab)

“Unfortunately, the study didn’t show a clinically relevant and statistically significant improvement on the primary neurocognitive endpoints, but the study confirmed the effects of tiratricol on reducing T3 concentrations, which are relevant to alleviate features of thyrotoxicosis in patients with MCT8 deficiency, regardless of age.” (opens the source at this quote in a new tab)