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.

Study Overview

• Status: Completed
• Conditions: Hereditary Tyrosinemia, Type I
• Intervention / Treatment: Drug: Nitisinone

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 Type: Interventional
• Enrollment (Actual): 18
• Phase: Phase 1

Contacts and Locations

Study Locations

• France
  • Paris, France
    • Hopital Necker
• Germany
  • Düsseldorf, Germany
    • Universitätsklinikum Düsseldorf
  • Heidelberg, Germany
    • Universitätsklinikum Heidelberg
  • München, Germany
    • Klinikum der Universität München
• United Kingdom
  • Birmingham, United Kingdom
    • Birmingham Children's Hospital
  • London, United Kingdom
    • Evelina Children's Hospital, St Thomas' Hospital
  • Manchester, United Kingdom
    • St Mary's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month to 17 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Orfadin suspension; Drug: nitisinone, oral suspension	Drug: Nitisinone; Oral suspension

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	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).	Day 3

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	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).	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).	Day 3

Collaborators and Investigators

• Sponsor: Swedish Orphan Biovitrum

Study record dates

Study Major Dates

• Study Start: October 1, 2012
• Primary Completion (Actual): March 1, 2013
• Study Completion (Actual): March 1, 2013

Study Registration Dates

• First Submitted: November 22, 2012
• First Submitted That Met QC Criteria: November 27, 2012
• First Posted (Estimate): November 28, 2012

Study Record Updates

• Last Update Posted (Estimate): November 6, 2014
• Last Update Submitted That Met QC Criteria: October 31, 2014
• Last Verified: October 1, 2014

More Information

Terms related to this study

• Keywords: taste; Orfadin; acceptability; suspension; nitisinone
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Tyrosinemias; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Nitisinone
• Other Study ID Numbers: Sobi.NTBC-002