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

January 13, 2020 updated by: Swedish Orphan Biovitrum

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Actual)

315

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Innsbruck, Austria
        • Swedish Orphan Biovitrum Investigational Site
      • Wien, Austria
        • Swedish Orphan Biovitrum Investigational Site
  • Belgium
      • 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, Belgium
        • Swedish Orphan Biovitrum Investigational Site
  • Czechia
      • Prague, Czechia
        • Swedish Orphan Biovitrum Investigational Site
  • Denmark
      • Copenhagen, Denmark
        • Swedish Orphan Biovitrum Investigational Site
  • Finland
      • Tampere, Finland
        • Swedish Orphan Biovitrum Investigational Site
      • Turku, Finland
        • Swedish Orphan Biovitrum Investigational Site
  • France
      • Angers, France
        • Swedish Orphan Biovitrum Investigational Site
      • Lille, France
        • Swedish Oprhan Biovitrum Investigational Site
      • Lyon, France
        • Swedish Orphan Biovitrum Investigational Site
      • Nantes, France
        • Swedish Orphan Biovitrum Investigational Site
      • Paris, France
        • Swedish Oprhan Biovitrum Investigational Site
      • Paris, France
        • Swedish Orphan Biovitrum Investigational Site
      • Talence, France
        • Swedish Orphan Biovitrum Investigational Site
  • Germany
      • 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, Germany
        • Swedish Orphan Biovitrum Investigational Site
  • Hungary
      • Budapest, Hungary
        • Swedish Orphan Biovitrum Investigational Site
      • Szeged, Hungary
        • Swedish Oprhan Biovitrum Investigational Site
  • Ireland
      • Dublin, Ireland
        • Swedish Orphan Biovitrum Investigational Site
  • Italy
      • 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, Italy
        • Swedish Orphan Biovitrum Investigational Site
  • Netherlands
      • Amsterdam, Netherlands
        • Swedish Oprhan Biovitrum Investigational Site
      • Utrecht, Netherlands
        • Swedish Orphan Biovitrum Investigational Site
  • Norway
      • Fredrikstad, Norway
        • Swedish Orphan Biovitrum Investigational Site
      • Levanger, Norway
        • Swedish Orphan Biovitrum Investigational Site
      • Oslo, Norway
        • Swedish Oprhan Biovitrum Investigational Site
      • Oslo, Norway
        • Swedish Orphan Biovitrum Investigational Site
      • Tromsö, Norway
        • Swedish Orphan Biovitrum Investigational Site
  • Poland
      • Warsaw, Poland
        • Swedish Oprhan Biovitrum Investigational Site
      • Warszawa, Poland
        • Swedish Orphan Biovitrum Investigational Site
  • Portugal
      • Coimbra, Portugal
        • Swedish Orphan Biovitrum Investigational Site
  • Spain
      • 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 Orphan Biovitrum Investigational Site
      • Madrid, Spain
        • Swedish Oprhan Biovitrum Investigational Site
      • Madrid, Spain
        • Swedish Orphan Biovitrum Investigational Site
      • Malaga, Spain
        • Swedish Orphan Biovitrum Investigational Site
      • Murcia, Spain
        • Swedish Orphan Biovitrum Investigational Site
      • Mérida, 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, Spain
        • Swedish Orphan Biovitrum Investigational Site
  • Sweden
      • Göteborg, Sweden
        • Swedish Orphan Biovitrum Investigational Site
      • Stockholm, Sweden, 14186
        • Swedish Orphan Biovitrum Investigational Site
      • Stockholm, Sweden
        • Swedish Orphan Biovitrum Investigational Site
      • Umeå, Sweden
        • Swedish Orphan Biovitrum Investigational Site
      • Uppsala, Sweden
        • Swedish Orphan Biovitrum Investigational Site
  • United Kingdom
      • Birmingham, United Kingdom
        • Swedish Oprhan Biovitrum Investigational Site
      • Bradford, United Kingdom
        • Swedish Orphan Biovitrum Investigational Site
      • London, United Kingdom
        • Swedish Oprhan Biovitrum Investigational Site
      • London, United Kingdom
        • Swedish Orphan Biovitrum Investigational Site
      • Manchester, United Kingdom
        • Swedish Oprhan Biovitrum Investigational Site
      • Manchester, United Kingdom
        • Swedish Orphan Biovitrum Investigational Site
      • Sheffield, United Kingdom
        • Swedish Orphan Biovitrum Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

All patients receiving Orfadin is eligible for the study

Description

Inclusion Criteria:

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

Exclusion Criteria:

  • No exclusion criteria

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Other

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
HT-1 patients on Orfadin treatment
HT-1 patients on Orfadin (nitisinone) treatment
Drug: Nitisinone
Nitisinone according to prescription
Other Names:
  • Orfadin

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Occurrence of Adverse events related to hepatic function
Time Frame: from 2005
Occurrence of Adverse events related to hepatic function
from 2005
Occurrence of Adverse events related to renal function
Time Frame: from 2005
Occurrence of Adverse events related to renal function
from 2005
Occurrence of Adverse events related to Ophthalmological function
Time Frame: from 2005
Occurrence of Adverse events related to Ophthalmological function
from 2005
Occurrence of Adverse events related to hematological function
Time Frame: from 2005
Occurrence of Adverse events related to hematological function
from 2005
Occurrence of Adverse events related to cognitive developmental function
Time Frame: from 2005
Occurrence of Adverse events related to cognitive developmental function
from 2005

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Occurrence of death
Time Frame: from 2005
Occurrence of death
from 2005
Occurrence of liver transplantation
Time Frame: from 2005
Occurrence of liver transplantation
from 2005
Occurrence of other Adverse Events
Time Frame: from 2005
Occurrence of Adverse Events other than those related to hepatic, renal, ophthalmic, hematological or cognitive functions
from 2005
Occurrence of discontinuation of Orfadin treatment
Time Frame: from 2005
Occurrence of discontinuation of Orfadin treatment
from 2005

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Swedish Orphan Biovitrum

Investigators

  • Study Director: Mattias Rudebeck, Swedish Orphan Biovitrum

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 1, 2013

Primary Completion (Actual)

September 30, 2019

Study Completion (Actual)

September 30, 2019

Study Registration Dates

First Submitted

October 23, 2014

First Submitted That Met QC Criteria

December 16, 2014

First Posted (Estimate)

December 19, 2014

Study Record Updates

Last Update Posted (Actual)

January 14, 2020

Last Update Submitted That Met QC Criteria

January 13, 2020

Last Verified

January 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Sobi.NTBC-005

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

IPD Plan Description

Discussions ongoing with regards to data sharing/registry.

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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