Clinical Trials Directory

Trials / Completed

CompletedNCT02320084

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

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

Status
Completed
Phase
—
Study type
Observational
Enrollment
315 (actual)
Sponsor
Swedish Orphan Biovitrum · Industry
Sex
All
Age
—
Healthy volunteers
Not accepted

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.

Conditions

Interventions

TypeNameDescription
DRUGNitisinoneNitisinone according to prescription

Timeline

Start date
2013-09-01
Primary completion
2019-09-30
Completion
2019-09-30
First posted
2014-12-19
Last updated
2024-09-19

Locations

77 sites across 17 countries: Austria, Belgium, Czechia, Denmark, Finland, France, Germany, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom

Source: ClinicalTrials.gov record NCT02320084. Inclusion in this directory is not an endorsement.