Taste and Palatability of Orfadin Suspension

Brief Title

Taste and Palatability of Orfadin Suspension

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.

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 Phase

Phase 1

Study Type

Interventional


Primary Outcome

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

Secondary Outcome

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

Condition

Hereditary Tyrosinemia, Type I

Intervention

Nitisinone

Study Arms / Comparison Groups

 Orfadin suspension
Description:  Drug: nitisinone, oral suspension

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

18

Start Date

October 2012

Completion Date

March 2013

Primary Completion Date

March 2013

Eligibility 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.
      

Gender

All

Ages

1 Month - 17 Years

Accepts Healthy Volunteers

No

Contacts

Erik Brouwer, MD, , 

Location Countries

France

Location Countries

France

Administrative Informations


NCT ID

NCT01734889

Organization ID

Sobi.NTBC-002


Responsible Party

Sponsor

Study Sponsor

Swedish Orphan Biovitrum


Study Sponsor

Erik Brouwer, MD, Study Director, Swedish Orphan Biovitrum


Verification Date

October 2014