Taste and Palatability of Orfadin Suspension

Swedish Orphan Biovitrum18 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hereditary Tyrosinemia, Type I

Interventions

NitisinoneDRUG

Oral suspension

Eligibility

Sex: ALLMin age: 1 MonthMax age: 17 Years

Medical Language ↔ Plain English

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.

Locations (7)

Hopital Necker

Paris, France

Universitätsklinikum Düsseldorf

Düsseldorf, Germany

Universitätsklinikum Heidelberg

Heidelberg, Germany

Klinikum der Universität München

München, Germany

Outcomes

Primary Outcomes

The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years

Patients rated the taste of the suspension. The following grading was applied: 5 (very good taste), 4 (good taste), 3 (neither good nor bad taste), 2 (bad taste) and 1 (very bad taste).

Time frame: Day 3

The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years

The parents of patients aged \<5 years rated their child´s acceptability of the suspension. The following grading was applied: 5 (very well), 4 (well), 3 (neither well nor badly), 2 (badly) and 1 (very badly).

Time frame: Day 3

Secondary Outcomes

The Palatability Scores on Day 1 (Subjects 5 - < 18 Years)

Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).

Time frame: Day 1

The Palatability Scores on Day 2 (Subjects 5 - < 18 Years)

Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).

Time frame: Day 2

The Palatability Scores on Day 3 (Subjects 5 - < 18 Years)

Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).

Time frame: Day 3

Data from ClinicalTrials.gov

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