Taste and Palatability of Orfadin Suspension

Study Overview
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Brief Summary
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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
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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.

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

Conditions
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Hereditary Tyrosinemia, Type I

Intervention / Treatment
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* Drug: Nitisinone


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 1 Month
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Orfadin suspension<br>Drug: nitisinone, oral suspension | Drug: Nitisinone<br>* Oral suspension<br>|

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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nitisinone, Orfadin, suspension, taste, acceptability