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Trial record 8 of 11 for:    "Tyrosinemia type 1"

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

Tracking Information
First Submitted Date  ICMJE November 22, 2012
First Posted Date  ICMJE November 28, 2012
Results First Submitted Date  ICMJE January 21, 2014
Results First Posted Date  ICMJE March 4, 2014
Last Update Posted Date November 6, 2014
Study Start Date  ICMJE October 2012
Actual Primary Completion Date March 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 21, 2014)
  • 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).
Original Primary Outcome Measures  ICMJE
 (submitted: November 27, 2012)
  • 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 ]
Change History Complete list of historical versions of study NCT01734889 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 21, 2014)
  • 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).
Original Secondary Outcome Measures  ICMJE
 (submitted: November 27, 2012)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Taste and Palatability of Orfadin Suspension
Official Title  ICMJE Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin.
Brief 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.
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.

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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 27, 2012)
18
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE March 2013
Actual Primary Completion Date March 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Month to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Germany,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01734889
Other Study ID Numbers  ICMJE Sobi.NTBC-002
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Swedish Orphan Biovitrum
Study Sponsor  ICMJE Swedish Orphan Biovitrum
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Erik Brouwer, MD Swedish Orphan Biovitrum
PRS Account Swedish Orphan Biovitrum
Verification Date October 2014

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