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

Study Overview
=================
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.

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

Conditions
-----------------
Hereditary Tyrosinemia, Type I

Intervention / Treatment
-----------------
* Drug: Nitisinone


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: All HT-1 patients receiving Orfadin treatment are eligible for entry. Exclusion Criteria: No exclusion criteria
Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| HT-1 patients on Orfadin treatment<br>HT-1 patients on Orfadin (nitisinone) treatment | Drug: Nitisinone<br>* Nitisinone according to prescription<br>* 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 | Occurrence of Adverse events related to hepatic function | from 2005 | 
| Occurrence of Adverse events related to renal function | Occurrence of Adverse events related to renal function | from 2005 | 
| Occurrence of Adverse events related to Ophthalmological function | Occurrence of Adverse events related to Ophthalmological function | from 2005 | 
| Occurrence of Adverse events related to hematological function | Occurrence of Adverse events related to hematological function | from 2005 | 
| Occurrence of Adverse events related to cognitive developmental function | Occurrence of Adverse events related to cognitive developmental function | from 2005 | 

Secondary Outcome Measures

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

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
HT-1, Hereditary Tyrosinemia, Type I, hypertyrosinemia type 1, Orfadin, Long term safety, Hereditary Tyrosinemia Type 1