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

NCT ID: NCT02320084

Last Updated: 2024-09-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

315 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-09-30

Study Completion Date

2019-09-30

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Hereditary Tyrosinemia, Type I

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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HT-1 patients on Orfadin treatment

HT-1 patients on Orfadin (nitisinone) treatment

Nitisinone

Intervention Type DRUG

Nitisinone according to prescription

Interventions

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Nitisinone

Nitisinone according to prescription

Intervention Type DRUG

Other Intervention Names

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Orfadin

Eligibility Criteria

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Inclusion Criteria

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

ALL

Accepts Healthy Volunteers

No

Sponsors

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Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Physician

Role: STUDY_DIRECTOR

Swedish Orphan Biovitrum

Locations

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Swedish Orphan Biovitrum Investigational Site

Innsbruck, , Austria

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Vienna, , Austria

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Antwerp, , Belgium

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Brussels, , Belgium

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Leuven, , Belgium

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Montegnée, , Belgium

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Woluwe-Saint-Lambert, , Belgium

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Prague, , Czechia

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Copenhagen, , Denmark

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Tampere, , Finland

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Turku, , Finland

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Angers, , France

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Lille, , France

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Lyon, , France

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Nantes, , France

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Paris, , France

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Paris, , France

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Talence, , France

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Düsseldorf, , Germany

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Erlangen, , Germany

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Freiburg im Breisgau, , Germany

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Hanover, , Germany

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Heidelberg, , Germany

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Leipzig, , Germany

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Magdeburg, , Germany

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München, , Germany

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Reutlingen, , Germany

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Budapest, , Hungary

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Szeged, , Hungary

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Dublin, , Ireland

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Bari, , Italy

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Catania, , Italy

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Florence, , Italy

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Milan, , Italy

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Monza, , Italy

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Napoli, , Italy

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Padua, , Italy

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Roma, , Italy

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Saliceto, , Italy

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Torino, , Italy

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Amsterdam, , Netherlands

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Utrecht, , Netherlands

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Fredrikstad, , Norway

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Levanger, , Norway

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Oslo, , Norway

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Oslo, , Norway

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Tromsø, , Norway

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Warsaw, , Poland

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Warsaw, , Poland

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Coimbra, , Portugal

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Albacete, , Spain

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Almería, , Spain

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Badalona, , Spain

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Barakaldo, , Spain

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Barcelona, , Spain

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Las Palmas de Gran Canaria, , Spain

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Madrid, , Spain

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Madrid, , Spain

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Málaga, , Spain

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Mérida, , Spain

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Murcia, , Spain

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Pamplona, , Spain

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Santiago, , Spain

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Seville, , Spain

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Valencia, , Spain

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Gothenburg, , Sweden

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Stockholm, , Sweden

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Stockholm, , Sweden

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Umeå, , Sweden

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Uppsala, , Sweden

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Birmingham, , United Kingdom

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Bradford, , United Kingdom

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London, , United Kingdom

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London, , United Kingdom

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Swedish Oprhan Biovitrum Investigational Site

Manchester, , United Kingdom

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Manchester, , United Kingdom

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Swedish Orphan Biovitrum Investigational Site

Sheffield, , United Kingdom

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Countries

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

Other Identifiers

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Sobi.NTBC-005

Identifier Type: -

Identifier Source: org_study_id

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