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

Brief Title

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

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.
    


Study Type

Observational


Primary Outcome

Occurrence of Adverse events related to hepatic function

Secondary Outcome

 Occurrence of death

Condition

Hereditary Tyrosinemia, Type I

Intervention

Nitisinone

Study Arms / Comparison Groups

 HT-1 patients on Orfadin treatment
Description:  HT-1 patients on Orfadin (nitisinone) treatment

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

315

Start Date

September 2013

Completion Date

September 30, 2019

Primary Completion Date

September 30, 2019

Eligibility Criteria

        Inclusion Criteria:

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

        Exclusion Criteria:

          -  No exclusion criteria
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Mattias Rudebeck, , 

Location Countries

Austria

Location Countries

Austria

Administrative Informations


NCT ID

NCT02320084

Organization ID

Sobi.NTBC-005


Responsible Party

Sponsor

Study Sponsor

Swedish Orphan Biovitrum


Study Sponsor

Mattias Rudebeck, Study Director, Swedish Orphan Biovitrum


Verification Date

January 2020