Taste and Palatability of Orfadin Suspension

Sobi

Status and phase

Completed

Phase 1

Conditions

Hereditary Tyrosinemia, Type I

Treatments

Drug: Nitisinone

Study type

Interventional

Funder types

Industry

Identifiers

NCT01734889

Sobi.NTBC-002

Details and patient eligibility

About

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.

Full 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.

Enrollment

18 patients

Sex

All

Ages

1 month to 17 years old

Volunteers

No Healthy Volunteers

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.