Conditions: Hereditary Tyrosinemia, Type I
Keywords: nitisinone Orfadin suspension taste acceptability
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 18
Healthy volunteers: no

Brief summary:
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.

Detailed description:
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.

Arms:
arm 1: Drug: nitisinone, oral suspension

Interventions: intervention 1: Nitisinone
Intervention descriptions: intervention 1: Oral suspension

Protocol document: