Taste and Palatability of Orfadin Suspension

Phase 1

Completed

• Conditions: Hereditary Tyrosinemia, Type I
• Interventions: Drug: Nitisinone

• Registration Number: NCT01734889
• Lead Sponsor: Swedish Orphan Biovitrum

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.

Study Timeline

• Study Start: 2012-10
• Study First Submitted: 2012-11-22
• Study First Submitted QC: 2012-11-27
• Study First Posted: 2012-11-28
• Primary Completion: 2013-03
• Study Completion: 2013-03
• Results First Submitted: 2014-01-21
• Results First Submitted QC: 2014-01-21
• Results First Posted: 2014-03-04
• Status Verified: 2014-10
• Last Update Submitted: 2014-10-31
• Last Update Posted: 2014-11-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 18

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Orfadin suspension	Nitisinone	Drug: nitisinone, oral suspension

Primary Outcome Measures

Name	Time	Method
The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years	Day 3	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).
The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years	Day 3	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).

Secondary Outcome Measures

Name	Time	Method
The Palatability Scores on Day 1 (Subjects 5 - < 18 Years)	Day 1	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).
The Palatability Scores on Day 2 (Subjects 5 - < 18 Years)	Day 2	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).
The Palatability Scores on Day 3 (Subjects 5 - < 18 Years)	Day 3	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).

Trial Locations

Locations (7)

Klinikum der Universität München; 🇩🇪 München, Germany

Hopital Necker; 🇫🇷 Paris, France

Universitätsklinikum Düsseldorf; 🇩🇪 Düsseldorf, Germany

Universitätsklinikum Heidelberg; 🇩🇪 Heidelberg, Germany

Birmingham Children's Hospital; 🇬🇧 Birmingham, United Kingdom

Evelina Children's Hospital, St Thomas' Hospital; 🇬🇧 London, United Kingdom

St Mary's Hospital; 🇬🇧 Manchester, United Kingdom