Hereditary Tyrosinemia, Type I Clinical Trial
Official title:
Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin.
The purpose of this study is to verify that pediatric patients, especially those who are not old enough to swallow capsules, accept the taste and palatability of a new suspension.
This is an open, non-randomized, non-controlled, multiple-dose study in 18 pediatric
patients. The treatment period is three days, and during the study the subjects will rate
the taste and palatability of the suspension or (for younger children) their parents will
rate the child´s acceptance of the suspension.
The study consists of a screening period, a 3 day treatment period and a 1 week follow-up
period.
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Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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