CompletedNot Applicable

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

Austria, Belgium315 participantsStarted 2013-09

Plain-language 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.

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * All HT-1 patients receiving Orfadin treatment are eligible for entry. Exclusion Criteria: * No exclusion criteria

What they're measuring

Occurrence of Adverse events related to hepatic function

Timeframe: from 2005

Occurrence of Adverse events related to renal function

Timeframe: from 2005

Occurrence of Adverse events related to Ophthalmological function

Timeframe: from 2005

Occurrence of Adverse events related to hematological function

Timeframe: from 2005

Occurrence of Adverse events related to cognitive developmental function

Timeframe: from 2005

Trial details

NCT IDNCT02320084

SponsorSwedish Orphan Biovitrum

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2019-09-30

View on ClinicalTrials.gov More Hereditary Tyrosinemia, Type I trials