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NCT Number: NCT02320084

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

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.

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Key information

Conditions

Hereditary Tyrosinemia, Type I Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Tyrosinemias

Sex eligibility

All sexes

Study type

Observational

Primary location

Swedish Orphan Biovitrum Investigational Site, Innsbruck, Austria

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • No exclusion criteria

Treatment and study plan

Nitisinone

Drug

Nitisinone according to prescription

Other names: Orfadin

Primary outcomes

  1. Occurrence of Adverse events related to hepatic function

    Time frame: from 2005

    Occurrence of Adverse events related to hepatic function

  2. Occurrence of Adverse events related to renal function

    Time frame: from 2005

    Occurrence of Adverse events related to renal function

  3. Occurrence of Adverse events related to Ophthalmological function

    Time frame: from 2005

    Occurrence of Adverse events related to Ophthalmological function

  4. Occurrence of Adverse events related to hematological function

    Time frame: from 2005

    Occurrence of Adverse events related to hematological function

  5. Occurrence of Adverse events related to cognitive developmental function

    Time frame: from 2005

    Occurrence of Adverse events related to cognitive developmental function

Secondary outcomes

  1. Occurrence of death

    Time frame: from 2005

    Occurrence of death

  2. Occurrence of liver transplantation

    Time frame: from 2005

    Occurrence of liver transplantation

  3. Occurrence of other Adverse Events

    Time frame: from 2005

    Occurrence of Adverse Events other than those related to hepatic, renal, ophthalmic, hematological or cognitive functions

  4. Occurrence of discontinuation of Orfadin treatment

    Time frame: from 2005

    Occurrence of discontinuation of Orfadin treatment

Sponsors and collaborators

Lead sponsor

Swedish Orphan Biovitrum

Industry

Registry information

Official study 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

Acronym: OPAL

Important dates

Study start
2013
Primary completion
2019
Study completion
2019
First posted
Dec 19, 2014
Registry last updated
Sep 19, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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