Hereditary Tyrosinemia, Type I
Conditions
Keywords
HT-1, Hereditary Tyrosinemia, Type I, hypertyrosinemia type 1, Orfadin, Long term safety, Hereditary Tyrosinemia Type 1
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.
Interventions
DRUGNitisinone
Nitisinone according to prescription
Sponsors
Swedish Orphan Biovitrum
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* All HT-1 patients receiving Orfadin treatment are eligible for entry.
Exclusion criteria
* No
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Occurrence of Adverse events related to cognitive developmental function | from 2005 | Occurrence of Adverse events related to cognitive developmental function |
| Occurrence of Adverse events related to hepatic function | from 2005 | Occurrence of Adverse events related to hepatic function |
| Occurrence of Adverse events related to renal function | from 2005 | Occurrence of Adverse events related to renal function |
| Occurrence of Adverse events related to Ophthalmological function | from 2005 | Occurrence of Adverse events related to Ophthalmological function |
| Occurrence of Adverse events related to hematological function | from 2005 | Occurrence of Adverse events related to hematological function |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Occurrence of liver transplantation | from 2005 | Occurrence of liver transplantation |
| Occurrence of other Adverse Events | from 2005 | Occurrence of Adverse Events other than those related to hepatic, renal, ophthalmic, hematological or cognitive functions |
| Occurrence of discontinuation of Orfadin treatment | from 2005 | Occurrence of discontinuation of Orfadin treatment |
| Occurrence of death | from 2005 | Occurrence of death |
Countries
Austria, Belgium, Czechia, Denmark, Finland, France, Germany, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
Outcome results
None listed