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Taste and Palatability of Orfadin Suspension

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ClinicalTrials.gov Identifier: NCT01734889
Recruitment Status : Completed
First Posted : November 28, 2012
Results First Posted : March 4, 2014
Last Update Posted : November 6, 2014
Sponsor:
Information provided by (Responsible Party):
Swedish Orphan Biovitrum

November 22, 2012
November 28, 2012
January 21, 2014
March 4, 2014
November 6, 2014
October 2012
March 2013   (Final data collection date for primary outcome measure)
  • 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).
  • 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).
  • The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years [ Time Frame: Day 0 to Day 3 ]
  • The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years [ Time Frame: Day 0 to Day 3 ]
Complete list of historical versions of study NCT01734889 on ClinicalTrials.gov Archive Site
  • 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).
  • 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).
  • 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).
  • The Palatability Scores on Day 1 (Subjects 5 - < 18 Years) [ Time Frame: Day 0 to Day 1 ]
  • The Palatability Scores on Day 2 (Subjects 5 - < 18 Years) [ Time Frame: Day 0 to Day 2 ]
  • The Palatability Scores on Day 3 (Subjects 5 - < 18 Years) [ Time Frame: Day 0 to Day 3 ]
Not Provided
Not Provided
 
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.

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.

Interventional
Phase 1
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Hereditary Tyrosinemia, Type I
Drug: Nitisinone
Oral suspension
Experimental: Orfadin suspension
Drug: nitisinone, oral suspension
Intervention: Drug: Nitisinone
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
18
Same as current
March 2013
March 2013   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
1 Month to 17 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
France,   Germany,   United Kingdom
 
 
NCT01734889
Sobi.NTBC-002
No
Not Provided
Not Provided
Swedish Orphan Biovitrum
Swedish Orphan Biovitrum
Not Provided
Study Director: Erik Brouwer, MD Swedish Orphan Biovitrum
Swedish Orphan Biovitrum
October 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP