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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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12 December 2020 |
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Main ID: |
NCT02320084 |
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Date of registration:
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23/10/2014 |
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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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Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
OPAL |
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Scientific title:
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A Non-interventional Post Authorization Study (PASS) to Evaluate Long-term Safety of Orfadin Treatment in Hypertyrosinemia Type 1 (HT-1) Patients in Standard Care |
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Date of first enrolment:
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September 2013 |
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Target sample size:
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315 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02320084 |
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Study type:
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Observational |
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Study design:
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Phase:
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Countries of recruitment
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Austria
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Belgium
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Croatia
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Czech Republic
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Czechia
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Denmark
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Finland
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France
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Germany
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Greece
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Hungary
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Ireland
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Italy
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Kosovo
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Lithuania
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Macedonia, The Former Yugoslav Republic of
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Netherlands
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Norway
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Poland
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Portugal
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Romania
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Slovakia
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Spain
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Sweden
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Switzerland
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United Kingdom
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Contacts
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Name:
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Mattias Rudebeck |
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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:
- All HT-1 patients receiving Orfadin treatment are eligible for entry.
Exclusion Criteria:
- No exclusion criteria
Age minimum:
N/A
Age maximum:
N/A
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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Occurrence of Adverse events related to hematological function
[Time Frame: from 2005]
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Occurrence of Adverse events related to Ophthalmological function
[Time Frame: from 2005]
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Occurrence of Adverse events related to renal function
[Time Frame: from 2005]
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Occurrence of Adverse events related to cognitive developmental function
[Time Frame: from 2005]
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Occurrence of Adverse events related to hepatic function
[Time Frame: from 2005]
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Secondary Outcome(s)
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Occurrence of liver transplantation
[Time Frame: from 2005]
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Occurrence of death
[Time Frame: from 2005]
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Occurrence of other Adverse Events
[Time Frame: from 2005]
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Occurrence of discontinuation of Orfadin treatment
[Time Frame: from 2005]
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Secondary ID(s)
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Sobi.NTBC-005
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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