Taste and Palatability of Orfadin Suspension

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Disease Information

Descriptive Information
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
Study Design † Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary Outcome Measure † The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 -
Secondary Outcome Measure † The Palatability Scores on Day 1 (Subjects 5 -
Condition † Hereditary Tyrosinemia, Type I
Intervention † DrugNitisinone
Study Arms / Comparison Groups Orfadin suspension 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 Both
Ages 1 Month - 17 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries † France
Administrative Information
NCT ID † NCT01734889
Organization ID Sobi.NTBC-002
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Swedish Orphan Biovitrum
Collaborators ††
Investigators † Study Director: Erik Brouwer, MD, Swedish Orphan Biovitrum
Information Provided By
Verification Date October 2014
First Received Date † November 22, 2012
Last Updated Date October 31, 2014
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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