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

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ClinicalTrials.gov Identifier: NCT02320084
Recruitment Status : Completed
First Posted : December 19, 2014
Last Update Posted : November 6, 2019
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
Hereditary Tyrosinemia, Type I Drug: Nitisinone

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.

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Study Type : Observational
Actual Enrollment : 315 participants
Observational Model: Cohort
Time Perspective: Other
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
Actual Study Start Date : September 2013
Actual Primary Completion Date : September 30, 2019
Actual Study Completion Date : September 30, 2019


Group/Cohort Intervention/treatment
HT-1 patients on Orfadin treatment
HT-1 patients on Orfadin (nitisinone) treatment
Drug: Nitisinone
Nitisinone according to prescription
Other Name: Orfadin




Primary Outcome Measures :
  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 Outcome Measures :
  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



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
All patients receiving Orfadin is eligible for the study
Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  • No exclusion criteria

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02320084


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Sponsors and Collaborators
Swedish Orphan Biovitrum
Investigators
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Study Director: Mattias Rudebeck Swedish Orphan Biovitrum

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Responsible Party: Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier: NCT02320084     History of Changes
Other Study ID Numbers: Sobi.NTBC-005
First Posted: December 19, 2014    Key Record Dates
Last Update Posted: November 6, 2019
Last Verified: November 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: Discussions ongoing with regards to data sharing/registry.
Keywords provided by Swedish Orphan Biovitrum:
HT-1
Hereditary Tyrosinemia, Type I
hypertyrosinemia type 1
Orfadin
Long term safety
Hereditary Tyrosinemia Type 1
Additional relevant MeSH terms:
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Tyrosinemias
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Nitisinone
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action