Effect of tiomolibdate choline on copper balance in patients with Wilson disease: An open-label phase 2 trial
An oral copper-reducing drug rapidly restores healthy copper balance in Wilson disease patients, expanding limited treatment options for this rare disorder.
In a phase 2 trial involving 9 Wilson disease patients, tiomolibdate choline (TMC) rapidly and significantly reduced daily copper balance with a meaningful -6.08 mg cumulative reduction over 21 days, driven by fecal excretion with an acceptable safety profile. These findings support the therapeutic potential of TMC as an oral copper-chelating agent in Wilson disease, an ultra-rare condition with limited current treatment options.
What the study was
- Study design
- Open-label single-arm phase 2 trial with Cu-balance metabolic study in clinical research unit
- Population
- Patients with Wilson disease (ultra-rare copper metabolism disorder)
- Sample size
- 9
- Category
- Treatment Innovation
- Maturity
- Exploratory
- Journal
- Hepatol Commun
Why it surfaced
Phase 2 trial demonstrating rapid, significant copper balance normalization in Wilson disease — an ultra-rare, often devastating disorder with limited treatment options. Small N tempered by ultra-rare disease context; population_unmet_need scored maximum per rare disease weighting rule.
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