Taste and Palatability of Orfadin Suspension

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-10-31

Study Completion Date

2013-03-31

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Hereditary Tyrosinemia, Type I

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Orfadin suspension

Drug: nitisinone, oral suspension

Group Type EXPERIMENTAL

Nitisinone

Intervention Type DRUG

Oral suspension

Interventions

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Nitisinone

Oral suspension

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

1 Month

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No