Results of the up-titration phase of the UNITED study, an open label, multicenter, prospective study to characterize the pharmacokinetics and pharmacodynamics of trientine dihydrochloride and to investigate the efficacy and safety in Wilson disease patients

Isabelle Mohr; Anna Członkowska; Piotr Socha; Aurélia Poujois; Aftab Ala; Thomas Damgaard Sandahl; Sanjay Bansal; Eduardo Couchonnal; Michael Praktiknjo; Constanze Leemhuis; C Kruse; Karl-Heinz Weiss; Anil Dhawan

doi:10.1055/s-0045-1810640

September 1, 2025Zeitschrift für Gastroenterologie

Results of the up-titration phase of the UNITED study, an open label, multicenter, prospective study to characterize the pharmacokinetics and pharmacodynamics of trientine dihydrochloride and to investigate the efficacy and safety in Wilson disease patients

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Key Points

The study assesses the pharmacokinetics and pharmacodynamics of trientine dihydrochloride, showing significant insights into efficacy.
Characterization of trientine dihydrochloride reveals safety profiles and implications for treatment in Wilson disease patients.
Open label, multicenter design provides robust data regarding dose-response relationships and the effects of trientine.
The findings support the established use of trientine in treating Wilson disease, highlighting its role in patient management.

Implication

Open label study evaluates trientine's efficacy and pharmacokinetics in Wilson disease patients, suggesting important safety profiles.

Abstract

Background: Trientine dihydrochloride (TETA-2HCl) has been used for the treatment of Wilson Disease for almost 40 years. Although its ability to chelate metal ions has been demonstrated in both non-clinical and clinical settings, no dedicated dose-response studies are available.

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Cite This Study

Mohr et al. (2025) studied this question.

synapsesocial.com/papers/68c194029b7b07f3a0618705 https://doi.org/10.1055/s-0045-1810640

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Results of the up-titration phase of the UNITED study, an open label, multicenter, prospective study to characterize the pharmacokinetics and pharmacodynamics of trientine dihydrochloride and to investigate the efficacy and safety in Wilson disease patients

Key Points

Implication

Abstract

Discussion

Cite This Study

Results of the up-titration phase of the UNITED study, an open label, multicenter, prospective study to characterize the pharmacokinetics and pharmacodynamics of trientine dihydrochloride and to investigate the efficacy and safety in Wilson disease patients

Key Points

Implication

Abstract

Discussion

Cite This Study

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