Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care (OPAL)
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02320084 |
Recruitment Status :
Completed
First Posted : December 19, 2014
Last Update Posted : January 14, 2020
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment |
---|---|
Hereditary Tyrosinemia, Type I | Drug: Nitisinone |
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 |
- Occurrence of Adverse events related to hepatic function [ Time Frame: from 2005 ]Occurrence of Adverse events related to hepatic function
- Occurrence of Adverse events related to renal function [ Time Frame: from 2005 ]Occurrence of Adverse events related to renal function
- Occurrence of Adverse events related to Ophthalmological function [ Time Frame: from 2005 ]Occurrence of Adverse events related to Ophthalmological function
- Occurrence of Adverse events related to hematological function [ Time Frame: from 2005 ]Occurrence of Adverse events related to hematological function
- Occurrence of Adverse events related to cognitive developmental function [ Time Frame: from 2005 ]Occurrence of Adverse events related to cognitive developmental function
- Occurrence of death [ Time Frame: from 2005 ]Occurrence of death
- Occurrence of liver transplantation [ Time Frame: from 2005 ]Occurrence of liver transplantation
- 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
- Occurrence of discontinuation of Orfadin treatment [ Time Frame: from 2005 ]Occurrence of discontinuation of Orfadin treatment

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
Inclusion Criteria:
- All HT-1 patients receiving Orfadin treatment are eligible for entry.
Exclusion Criteria:
- No exclusion criteria

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

Study Director: | Mattias Rudebeck | Swedish Orphan Biovitrum |
Responsible Party: | Swedish Orphan Biovitrum |
ClinicalTrials.gov Identifier: | NCT02320084 |
Other Study ID Numbers: |
Sobi.NTBC-005 |
First Posted: | December 19, 2014 Key Record Dates |
Last Update Posted: | January 14, 2020 |
Last Verified: | January 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Plan Description: | Discussions ongoing with regards to data sharing/registry. |
HT-1 Hereditary Tyrosinemia, Type I hypertyrosinemia type 1 |
Orfadin Long term safety Hereditary Tyrosinemia Type 1 |
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 |