Taste and Palatability of Orfadin Suspension

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier:
NCT01734889
First received: November 22, 2012
Last updated: March 7, 2014
Last verified: March 2014
  Purpose

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.


Condition Intervention Phase
Hereditary Tyrosinemia Type 1 (HT-1)
Drug: Nitisinone
Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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.

Resource links provided by NLM:


Further study details as provided by Swedish Orphan Biovitrum:

Primary Outcome Measures:
  • The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years [ Time Frame: Day 3 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Day 3 ] [ Designated as safety issue: No ]
    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:
  • The Palatability Scores on Day 1 (Subjects 5 - < 18 Years) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
    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) [ Time Frame: Day 2 ] [ Designated as safety issue: No ]
    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) [ Time Frame: Day 3 ] [ Designated as safety issue: No ]
    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).


Enrollment: 18
Study Start Date: October 2012
Study Completion Date: March 2013
Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Orfadin suspension
Drug: nitisinone, oral suspension
Drug: Nitisinone
Oral 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.

  Eligibility

Ages Eligible for Study:   1 Month to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01734889

Locations
France
Hopital Necker
Paris, France
Germany
Universitätsklinikum Düsseldorf
Düsseldorf, Germany
Universitätsklinikum Heidelberg
Heidelberg, Germany
Klinikum der Universität München
München, Germany
United Kingdom
Birmingham Children's Hospital
Birmingham, United Kingdom
Evelina Children's Hospital, St Thomas' Hospital
London, United Kingdom
St Mary's Hospital
Manchester, United Kingdom
Sponsors and Collaborators
Swedish Orphan Biovitrum
Investigators
Study Director: Erik Brouwer, MD Swedish Orphan Biovitrum
  More Information

No publications provided

Responsible Party: Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier: NCT01734889     History of Changes
Other Study ID Numbers: Sobi.NTBC-002
Study First Received: November 22, 2012
Results First Received: January 21, 2014
Last Updated: March 7, 2014
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
Germany: Federal Institute for Drugs and Medical Devices
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by Swedish Orphan Biovitrum:
nitisinone
Orfadin
suspension
taste
acceptability

Additional relevant MeSH terms:
Tyrosinemias
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Nitisinone
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 15, 2014