Clinical trial NCT02320084: Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
Official title: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hereditary Tyrosinemia, Type I
Keywords: HT-1
Hereditary Tyrosinemia, Type I
hypertyrosinemia type 1
Orfadin
Long term safety
Hereditary Tyrosinemia Type 1
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
First posted: 2014-12-19
Enrollment: 315
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients receiving Orfadin is eligible for the study

Brief summary: The purpose of this study is to look at the long term safety profile of Orfadin treatment in patients suffering from hereditary tyrosinemia type 1 (HT-1). Patients included in the study will use Orfadin according to normal clinical practice.

Detailed description: The planned study is a non-interventional study that will look at the long-term safety of Orfadin treatment in patients suffering from hereditary tyrosinemia type 1. Orfadin will be used according to normal practice . There is an ongoing post-marketing surveillance (PMS) program to monitor hepatic, renal, hematological, neurological and ophthalmic status in all patients treated with Orfadin. The Committee for medicinal Products for Human Use (CHMP) has required this program and have looked at the data for approximately 400 patients and found the benefit-risk ratio to be positive. The present study (PASS) will replace the ongoing PMS. The transition of countries will be gradual; starting in 2013.The study will include HT-1 patients on Orfadin treatment in standard clinical care as well as newly diagnosed patients just starting with Orfadin treatment.

Eligibility criteria: Inclusion Criteria:

* All HT-1 patients receiving Orfadin treatment are eligible for entry.

Exclusion Criteria:

* No exclusion criteria

Primary outcomes:
measure: Occurrence of Adverse events related to hepatic function
timeFrame: from 2005
measure: Occurrence of Adverse events related to renal function
timeFrame: from 2005
measure: Occurrence of Adverse events related to Ophthalmological function
timeFrame: from 2005
measure: Occurrence of Adverse events related to hematological function
timeFrame: from 2005
measure: Occurrence of Adverse events related to cognitive developmental function
timeFrame: from 2005
Secondary outcomes:
measure: Occurrence of death
timeFrame: from 2005
measure: Occurrence of liver transplantation
timeFrame: from 2005
measure: Occurrence of other Adverse Events
timeFrame: from 2005
measure: Occurrence of discontinuation of Orfadin treatment
timeFrame: from 2005
Other outcomes:
Locations:
city: Innsbruck
country: Austria
facility: Swedish Orphan Biovitrum Investigational Site
city: Wien
country: Austria
facility: Swedish Orphan Biovitrum Investigational Site
city: Antwerpen
country: Belgium
facility: Swedish Orphan Biovitrum Investigational Site
city: Brussels
country: Belgium
facility: Swedish Orphan Biovitrum Investigational Site
city: Leuven
country: Belgium
facility: Swedish Orphan Biovitrum Investigational Site
city: Montegnée
country: Belgium
facility: Swedish Orphan Biovitrum Investigational Site
city: Woluwe-Saint-Lambert
country: Belgium
facility: Swedish Orphan Biovitrum Investigational Site
city: Prague
country: Czechia
facility: Swedish Orphan Biovitrum Investigational Site
city: Copenhagen
country: Denmark
facility: Swedish Orphan Biovitrum Investigational Site
city: Tampere
country: Finland
facility: Swedish Orphan Biovitrum Investigational Site
city: Turku
country: Finland
facility: Swedish Orphan Biovitrum Investigational Site
city: Angers
country: France
facility: Swedish Orphan Biovitrum Investigational Site
city: Lille
country: France
facility: Swedish Oprhan Biovitrum Investigational Site
city: Lyon
country: France
facility: Swedish Orphan Biovitrum Investigational Site
city: Nantes
country: France
facility: Swedish Orphan Biovitrum Investigational Site
city: Paris
country: France
facility: Swedish Oprhan Biovitrum Investigational Site
city: Paris
country: France
facility: Swedish Orphan Biovitrum Investigational Site
city: Talence
country: France
facility: Swedish Orphan Biovitrum Investigational Site
city: Düsseldorf
country: Germany
facility: Swedish Orphan Biovitrum Investigational Site
city: Erlangen
country: Germany
facility: Swedish Orphan Biovitrum Investigational Site
city: Freiburg
country: Germany
facility: Swedish Orphan Biovitrum Investigational Site
city: Hannover
country: Germany
facility: Swedish Orphan Biovitrum Investigational Site
city: Heidelberg
country: Germany
facility: Swedish Orphan Biovitrum Investigational Site
city: Leipzig
country: Germany
facility: Swedish Orphan Biovitrum Investigational Site
city: Magdeburg
country: Germany
facility: Swedish Orphan Biovitrum Investigational Site
city: München
country: Germany
facility: Swedish Orphan Biovitrum Investigational Site
city: Reutlingen
country: Germany
facility: Swedish Orphan Biovitrum Investigational Site
city: Budapest
country: Hungary
facility: Swedish Orphan Biovitrum Investigational Site
city: Szeged
country: Hungary
facility: Swedish Oprhan Biovitrum Investigational Site
city: Dublin
country: Ireland
facility: Swedish Orphan Biovitrum Investigational Site
city: Bari
country: Italy
facility: Swedish Orphan Biovitrum Investigational Site
city: Catania
country: Italy
facility: Swedish Orphan Biovitrum Investigational Site
city: Firenze
country: Italy
facility: Swedish Orphan Biovitrum Investigational Site
city: Milano
country: Italy
facility: Swedish Orphan Biovitrum Investigational Site
city: Monza
country: Italy
facility: Swedish Orphan Biovitrum Investigational Site
city: Napoli
country: Italy
facility: Swedish Orphan Biovitrum Investigational Site
city: Padova
country: Italy
facility: Swedish Orphan Biovitrum Investigational Site
city: Roma
country: Italy
facility: Swedish Orphan Biovitrum Investigational Site
city: Saliceto
country: Italy
facility: Swedish Orphan Biovitrum Investigational Site
city: Torino
country: Italy
facility: Swedish Orphan Biovitrum Investigational Site
city: Amsterdam
country: Netherlands
facility: Swedish Oprhan Biovitrum Investigational Site
city: Utrecht
country: Netherlands
facility: Swedish Orphan Biovitrum Investigational Site
city: Fredrikstad
country: Norway
facility: Swedish Orphan Biovitrum Investigational Site
city: Levanger
country: Norway
facility: Swedish Orphan Biovitrum Investigational Site
city: Oslo
country: Norway
facility: Swedish Oprhan Biovitrum Investigational Site
city: Oslo
country: Norway
facility: Swedish Orphan Biovitrum Investigational Site
city: Tromsö
country: Norway
facility: Swedish Orphan Biovitrum Investigational Site
city: Warsaw
country: Poland
facility: Swedish Oprhan Biovitrum Investigational Site
city: Warszawa
country: Poland
facility: Swedish Orphan Biovitrum Investigational Site
city: Coimbra
country: Portugal
facility: Swedish Orphan Biovitrum Investigational Site
city: Albacete
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Almeria
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Badalona
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Barakaldo
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Barcelona
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Las Palmas de Gran Canaria
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Madrid
country: Spain
facility: Swedish Oprhan Biovitrum Investigational Site
city: Madrid
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Malaga
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Murcia
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Mérida
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Pamplona
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Santiago
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Sevilla
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Valencia
country: Spain
facility: Swedish Orphan Biovitrum Investigational Site
city: Göteborg
country: Sweden
facility: Swedish Orphan Biovitrum Investigational Site
city: Stockholm
country: Sweden
facility: Swedish Orphan Biovitrum Investigational Site
city: Stockholm
country: Sweden
facility: Swedish Orphan Biovitrum Investigational Site
city: Umeå
country: Sweden
facility: Swedish Orphan Biovitrum Investigational Site
city: Uppsala
country: Sweden
facility: Swedish Orphan Biovitrum Investigational Site
city: Birmingham
country: United Kingdom
facility: Swedish Oprhan Biovitrum Investigational Site
city: Bradford
country: United Kingdom
facility: Swedish Orphan Biovitrum Investigational Site
city: London
country: United Kingdom
facility: Swedish Oprhan Biovitrum Investigational Site
city: London
country: United Kingdom
facility: Swedish Orphan Biovitrum Investigational Site
city: Manchester
country: United Kingdom
facility: Swedish Oprhan Biovitrum Investigational Site
city: Manchester
country: United Kingdom
facility: Swedish Orphan Biovitrum Investigational Site
city: Sheffield
country: United Kingdom
facility: Swedish Orphan Biovitrum Investigational Site
Study officials:
affiliation: Swedish Orphan Biovitrum
name: Study Physician
role: STUDY_DIRECTOR
References: