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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

315

participants targeted

Target at P75+ for all trials

Timeline

Completed

Started Sep 2013

Longer than P75 for all trials

Geographic Reach

17 countries

77 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

6.1 years study duration

Study Start

First participant enrolled

September 1, 2013

Completed

1.1 years until next milestone

First Submitted

Initial submission to the registry

October 23, 2014

Completed

2 months until next milestone

First Posted

Study publicly available on registry

December 19, 2014

Completed

4.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2019

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2019

Completed

Last Updated

September 19, 2024

Status Verified

September 1, 2024

Enrollment Period

6.1 years

First QC Date

October 23, 2014

Last Update Submit

September 17, 2024

Conditions

Hereditary Tyrosinemia, Type I

Keywords

HT-1Hereditary Tyrosinemia, Type Ihypertyrosinemia type 1OrfadinLong term safetyHereditary Tyrosinemia Type 1

Outcome Measures

Primary Outcomes (5)

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 Outcomes (4)

Occurrence of death
from 2005
Occurrence of liver transplantation
from 2005
Occurrence of other Adverse Events
from 2005
Occurrence of discontinuation of Orfadin treatment
from 2005

Study Arms (1)

HT-1 patients on Orfadin treatment

HT-1 patients on Orfadin (nitisinone) treatment

Drug: Nitisinone

Interventions

NitisinoneDRUG

Nitisinone according to prescription

Also known as: Orfadin

HT-1 patients on Orfadin treatment

Eligibility Criteria

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodNon-Probability Sample

Study Population

All patients receiving Orfadin is eligible for the study

You may qualify if:

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

Contact the study team to confirm eligibility.