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