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

Completed

• Conditions: Hereditary Tyrosinemia, Type I
• Interventions: Drug: Nitisinone

• Registration Number: NCT02320084
• Lead Sponsor: Swedish Orphan Biovitrum

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 Timeline

• Study Start: 2013-09
• Study First Submitted: 2014-10-23
• Study First Submitted QC: 2014-12-16
• Study First Posted: 2014-12-19
• Primary Completion: 2019-09-30
• Study Completion: 2019-09-30
• Status Verified: 2020-01
• Last Update Submitted: 2020-01-13
• Last Update Posted: 2020-01-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 315

Inclusion Criteria

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

Exclusion Criteria

• No exclusion criteria

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
HT-1 patients on Orfadin treatment	Nitisinone	HT-1 patients on Orfadin (nitisinone) treatment

Primary Outcome Measures

Name	Time	Method
Occurrence of Adverse events related to renal function	from 2005	Occurrence of Adverse events related to renal 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 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
Occurrence of Adverse events related to cognitive developmental function	from 2005	Occurrence of Adverse events related to cognitive developmental function

Secondary Outcome Measures

Name	Time	Method
Occurrence of liver transplantation	from 2005	Occurrence of liver transplantation
Occurrence of discontinuation of Orfadin treatment	from 2005	Occurrence of discontinuation of Orfadin treatment
Occurrence of death	from 2005	Occurrence of death
Occurrence of other Adverse Events	from 2005	Occurrence of Adverse Events other than those related to hepatic, renal, ophthalmic, hematological or cognitive functions

Trial Locations

Locations (2)

Swedish Orphan Biovitrum Investigational Site; 🇬🇧 Sheffield, United Kingdom

Swedish Oprhan Biovitrum Investigational Site; 🇬🇧 Manchester, United Kingdom