Taste and Palatability of Orfadin Suspension

Hereditary Tyrosinemia, Type I Clinical Trial

Official title:

Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01734889
• Other study ID #: Sobi.NTBC-002
• Status: Completed
• Phase: Phase 1
• First received: November 22, 2012
• Last updated: October 31, 2014
• Start date: October 2012
• Est. completion date: March 2013

Study information

• Verified date: October 2014
• Source: Swedish Orphan Biovitrum
• Contact: n/a
• Is FDA regulated: No
• Health authority: United Kingdom: Medicines and Healthcare Products Regulatory AgencyGermany: Federal Institute for Drugs and Medical DevicesFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: March 2013
• Est. primary completion date: March 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Month to 17 Years

Eligibility

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

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hereditary Tyrosinemia, Type I
• Tyrosinemias

Intervention

Drug:
• Nitisinone
Oral suspension

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Swedish Orphan Biovitrum

Countries where clinical trial is conducted

France,  Germany,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Primary	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	No
Secondary	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	No
Secondary	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	No
Secondary	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	No