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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 October 2017 |
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Main ID: |
NCT01734889 |
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Date of registration:
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22/11/2012 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Taste and Palatability of Orfadin Suspension
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Scientific title:
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Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin. |
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Date of first enrolment:
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October 2012 |
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Target sample size:
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18 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01734889 |
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Study type:
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Interventional |
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Study design:
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Phase:
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Phase 1
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Countries of recruitment
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France
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Germany
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United Kingdom
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Contacts
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Name:
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Erik Brouwer, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Swedish Orphan Biovitrum |
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Key inclusion & exclusion 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.
Age minimum:
1 Month
Age maximum:
17 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Hereditary Tyrosinemia, Type I
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Intervention(s)
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Drug: Nitisinone
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Primary Outcome(s)
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The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years
[Time Frame: Day 3]
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The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years
[Time Frame: Day 3]
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Secondary Outcome(s)
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The Palatability Scores on Day 1 (Subjects 5 - < 18 Years)
[Time Frame: Day 1]
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The Palatability Scores on Day 3 (Subjects 5 - < 18 Years)
[Time Frame: Day 3]
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The Palatability Scores on Day 2 (Subjects 5 - < 18 Years)
[Time Frame: Day 2]
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Secondary ID(s)
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Sobi.NTBC-002
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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