Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care

Hereditary Tyrosinemia, Type I Clinical Trial

— OPAL  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02320084
• Other study ID #: Sobi.NTBC-005
• Status: Completed
• Start date: September 2013
• Est. completion date: September 30, 2019

Study information

• Verified date: January 2020
• Source: Swedish Orphan Biovitrum
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 315
• Est. completion date: September 30, 2019
• Est. primary completion date: September 30, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

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

Exclusion Criteria:

• No exclusion criteria

Related Conditions & MeSH terms

• Hereditary Tyrosinemia, Type I
• Tyrosinemias

Intervention

Drug:
• Nitisinone
Nitisinone according to prescription

Locations

Country	Name	City	State
Austria	Swedish Orphan Biovitrum Investigational Site	Innsbruck
Austria	Swedish Orphan Biovitrum Investigational Site	Wien
Belgium	Swedish Orphan Biovitrum Investigational Site	Antwerpen
Belgium	Swedish Orphan Biovitrum Investigational Site	Brussels
Belgium	Swedish Orphan Biovitrum Investigational Site	Leuven
Belgium	Swedish Orphan Biovitrum Investigational Site	Montegnée
Belgium	Swedish Orphan Biovitrum Investigational Site	Woluwe-Saint-Lambert
Czechia	Swedish Orphan Biovitrum Investigational Site	Prague
Denmark	Swedish Orphan Biovitrum Investigational Site	Copenhagen
Finland	Swedish Orphan Biovitrum Investigational Site	Tampere
Finland	Swedish Orphan Biovitrum Investigational Site	Turku
France	Swedish Orphan Biovitrum Investigational Site	Angers
France	Swedish Oprhan Biovitrum Investigational Site	Lille
France	Swedish Orphan Biovitrum Investigational Site	Lyon
France	Swedish Orphan Biovitrum Investigational Site	Nantes
France	Swedish Oprhan Biovitrum Investigational Site	Paris
France	Swedish Orphan Biovitrum Investigational Site	Paris
France	Swedish Orphan Biovitrum Investigational Site	Talence
Germany	Swedish Orphan Biovitrum Investigational Site	Düsseldorf
Germany	Swedish Orphan Biovitrum Investigational Site	Erlangen
Germany	Swedish Orphan Biovitrum Investigational Site	Freiburg
Germany	Swedish Orphan Biovitrum Investigational Site	Hannover
Germany	Swedish Orphan Biovitrum Investigational Site	Heidelberg
Germany	Swedish Orphan Biovitrum Investigational Site	Leipzig
Germany	Swedish Orphan Biovitrum Investigational Site	Magdeburg
Germany	Swedish Orphan Biovitrum Investigational Site	München
Germany	Swedish Orphan Biovitrum Investigational Site	Reutlingen
Hungary	Swedish Orphan Biovitrum Investigational Site	Budapest
Hungary	Swedish Oprhan Biovitrum Investigational Site	Szeged
Ireland	Swedish Orphan Biovitrum Investigational Site	Dublin
Italy	Swedish Orphan Biovitrum Investigational Site	Bari
Italy	Swedish Orphan Biovitrum Investigational Site	Catania
Italy	Swedish Orphan Biovitrum Investigational Site	Firenze
Italy	Swedish Orphan Biovitrum Investigational Site	Milano
Italy	Swedish Orphan Biovitrum Investigational Site	Monza
Italy	Swedish Orphan Biovitrum Investigational Site	Napoli
Italy	Swedish Orphan Biovitrum Investigational Site	Padova
Italy	Swedish Orphan Biovitrum Investigational Site	Roma
Italy	Swedish Orphan Biovitrum Investigational Site	Saliceto
Italy	Swedish Orphan Biovitrum Investigational Site	Torino
Netherlands	Swedish Oprhan Biovitrum Investigational Site	Amsterdam
Netherlands	Swedish Orphan Biovitrum Investigational Site	Utrecht
Norway	Swedish Orphan Biovitrum Investigational Site	Fredrikstad
Norway	Swedish Orphan Biovitrum Investigational Site	Levanger
Norway	Swedish Oprhan Biovitrum Investigational Site	Oslo
Norway	Swedish Orphan Biovitrum Investigational Site	Oslo
Norway	Swedish Orphan Biovitrum Investigational Site	Tromsö
Poland	Swedish Oprhan Biovitrum Investigational Site	Warsaw
Poland	Swedish Orphan Biovitrum Investigational Site	Warszawa
Portugal	Swedish Orphan Biovitrum Investigational Site	Coimbra
Spain	Swedish Orphan Biovitrum Investigational Site	Albacete
Spain	Swedish Orphan Biovitrum Investigational Site	Almeria
Spain	Swedish Orphan Biovitrum Investigational Site	Badalona
Spain	Swedish Orphan Biovitrum Investigational Site	Barakaldo
Spain	Swedish Orphan Biovitrum Investigational Site	Barcelona
Spain	Swedish Orphan Biovitrum Investigational Site	Las Palmas de Gran Canaria
Spain	Swedish Oprhan Biovitrum Investigational Site	Madrid
Spain	Swedish Orphan Biovitrum Investigational Site	Madrid
Spain	Swedish Orphan Biovitrum Investigational Site	Malaga
Spain	Swedish Orphan Biovitrum Investigational Site	Mérida
Spain	Swedish Orphan Biovitrum Investigational Site	Murcia
Spain	Swedish Orphan Biovitrum Investigational Site	Pamplona
Spain	Swedish Orphan Biovitrum Investigational Site	Santiago
Spain	Swedish Orphan Biovitrum Investigational Site	Sevilla
Spain	Swedish Orphan Biovitrum Investigational Site	Valencia
Sweden	Swedish Orphan Biovitrum Investigational Site	Göteborg
Sweden	Swedish Orphan Biovitrum Investigational Site	Stockholm
Sweden	Swedish Orphan Biovitrum Investigational Site	Stockholm
Sweden	Swedish Orphan Biovitrum Investigational Site	Umeå
Sweden	Swedish Orphan Biovitrum Investigational Site	Uppsala
United Kingdom	Swedish Oprhan Biovitrum Investigational Site	Birmingham
United Kingdom	Swedish Orphan Biovitrum Investigational Site	Bradford
United Kingdom	Swedish Oprhan Biovitrum Investigational Site	London
United Kingdom	Swedish Orphan Biovitrum Investigational Site	London
United Kingdom	Swedish Oprhan Biovitrum Investigational Site	Manchester
United Kingdom	Swedish Orphan Biovitrum Investigational Site	Manchester
United Kingdom	Swedish Orphan Biovitrum Investigational Site	Sheffield

Sponsors (1)

Lead Sponsor	Collaborator
Swedish Orphan Biovitrum

Countries where clinical trial is conducted

Austria,  Belgium,  Czechia,  Denmark,  Finland,  France,  Germany,  Hungary,  Ireland,  Italy,  Netherlands,  Norway,  Poland,  Portugal,  Spain,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Occurrence of Adverse events related to hepatic function	Occurrence of Adverse events related to hepatic function	from 2005
Primary	Occurrence of Adverse events related to renal function	Occurrence of Adverse events related to renal function	from 2005
Primary	Occurrence of Adverse events related to Ophthalmological function	Occurrence of Adverse events related to Ophthalmological function	from 2005
Primary	Occurrence of Adverse events related to hematological function	Occurrence of Adverse events related to hematological function	from 2005
Primary	Occurrence of Adverse events related to cognitive developmental function	Occurrence of Adverse events related to cognitive developmental function	from 2005
Secondary	Occurrence of death	Occurrence of death	from 2005
Secondary	Occurrence of liver transplantation	Occurrence of liver transplantation	from 2005
Secondary	Occurrence of other Adverse Events	Occurrence of Adverse Events other than those related to hepatic, renal, ophthalmic, hematological or cognitive functions	from 2005
Secondary	Occurrence of discontinuation of Orfadin treatment	Occurrence of discontinuation of Orfadin treatment	from 2005