Taste and palatability of Orfadin suspension.

Conditions: Hereditary tyrosinemia type 1 (HT-1)
MedDRA version: 14.1Level: LLTClassification code 10069459Term: Tyrosinaemia type ISystem Organ Class: 100000004850
Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Registration Number: EUCTR2012-002286-36-DE

Lead Sponsor: Swedish Orphan Biovitrum AB

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 18

Inclusion Criteria

Patients with HT-1 currently managed on Orfadin (nitisinone) capsules.
Age from 1 month to less than 18 years.
Signed informed consent.
Are the trial subjects under 18? yes
Number of subjects for this age range: 18
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To determine the acceptability of the suspension in the pediatric population.;Secondary Objective: To assess the taste of the suspension on Days 1 and 2 for subjects 5- < 18 years.<br>To assess the acceptability of the suspension on Days 1 and 2 for subjects < 5years.<br>To assess the palatability of the suspension on Days 1, 2 and 3 for subjects 5- < 18 years.<br>To assess the overall acceptability of the suspension in subjects swallowing the capsules whole and subjects mixing capsule contents with food, respectively.<br>To assess the tolerability of the suspension.;Primary end point(s): The taste score for the last dose of the suspension on Day 3 for subjects 5 -< 18 years.<br>The acceptability score for the last dose of the suspension on Day 3 for subjects < 5 years.;Timepoint(s) of evaluation of this end point: Day 3

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Taste scores on Days 1 and 2 (subjects 5-<18 years).<br>The acceptability scores on Days 1 and 2 (subjects < 5 years).<br>The palatability scores on Days 1, 2 and 3 (subjects 5-<18 years).<br>The overall acceptability response (subjects 0-<18 years).<br>Adverse events.;Timepoint(s) of evaluation of this end point: Days 1, 2 and 3.