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

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Disease Information

Descriptive Information
Brief Title † Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
Official 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
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 Phase N/A
Study Type † Observational
Study Design †
Primary Outcome Measure † Occurrence of Adverse events related to hepatic function
Secondary Outcome Measure † Occurrence of death
Condition † Hereditary Tyrosinemia, Type I
Intervention † DrugNitisinone
Study Arms / Comparison Groups HT-1 patients on Orfadin treatment HT-1 patients on Orfadin (nitisinone) treatment
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 400
Start Date † September 2013
Completion Date September 2019
Primary Completion Date September 2019
Eligibility Criteria † Inclusion Criteria: - All HT-1 patients receiving Orfadin treatment are eligible for entry. Exclusion Criteria: - No exclusion criteria
Gender All
Ages N/A - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † Austria
Administrative Information
NCT ID † NCT02320084
Organization ID Sobi.NTBC-005
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Swedish Orphan Biovitrum
Collaborators ††
Investigators † Study Director: Anders Bröijersén, MD, PhD, Swedish Orphan Biovitrum
Information Provided By
Verification Date May 2017
First Received Date † October 23, 2014
Last Updated Date May 29, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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