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Completed

NCT Number: NCT01734889

Taste and Palatability of Orfadin Suspension

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.

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Key information

Conditions

Hereditary Tyrosinemia, Type I Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Tyrosinemias

Age range

1 month–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Hopital Necker, Paris, France

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Nitisinone

Drug

Oral suspension

Primary outcomes

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

    Time frame: 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).

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

    Time frame: 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 outcomes

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

    Time frame: 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).

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

    Time frame: 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).

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

    Time frame: 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).

Sponsors and collaborators

Lead sponsor

Swedish Orphan Biovitrum

Industry

Registry information

Official study 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.

Important dates

Study start
2012
Primary completion
2013
Study completion
2013
First posted
Nov 28, 2012
Registry last updated
Nov 6, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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