Taste and Palatability of Orfadin Suspension

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.

Inclusion Criteria:

• Patients with HT-1 currently managed on Orfadin (nitisinone) capsules.
• Age from 1 month to less than 18 years.
• Signed informed consent.

Exclusion Criteria:

• Any medical condition which in the opinion of the investigator makes the subject unsuitable for inclusion.
• Enrollment in another concurrent clinical study, or intake of an investigational medicinal product (IMP), within one month prior to inclusion in this study.
• Foreseeable inability to cooperate with given instructions or study procedures.