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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
Sponsor:
Information provided by (Responsible Party):
Swedish Orphan Biovitrum

Tracking Information
First Submitted Date October 23, 2014
First Posted Date December 19, 2014
Last Update Posted Date January 14, 2020
Actual Study Start Date September 2013
Actual Primary Completion Date September 30, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 29, 2017)
  • 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
Original Primary Outcome Measures
 (submitted: December 16, 2014)
  • Occurrence of Adverse events related to hepatic function [ Time Frame: 4 years ]
  • Occurrence of Adverse events related to renal function [ Time Frame: 4 years ]
  • Occurrence of Adverse events related to Ophthalmological function [ Time Frame: 4 years ]
  • Occurrence of Adverse events related to hematological function [ Time Frame: 4 years ]
  • Occurrence of Adverse events related to cognitive developmental function [ Time Frame: 4 years ]
Change History
Current Secondary Outcome Measures
 (submitted: May 29, 2017)
  • 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
Original Secondary Outcome Measures
 (submitted: December 16, 2014)
  • Occurrence of death [ Time Frame: 4 years ]
  • Occurrence of liver transplantation [ Time Frame: 4 years ]
  • Occurrence of other Adverse Events [ Time Frame: 4 years ]
    Occurrence of AEs other than those related to hepatic, renal, ophthalmic, hematological or cognitive functions
  • Occurrence of discontinuation of Orfadin treatment [ Time Frame: 4 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
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 Type Observational
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population All patients receiving Orfadin is eligible for the study
Condition Hereditary Tyrosinemia, Type I
Intervention Drug: Nitisinone
Nitisinone according to prescription
Other Name: Orfadin
Study Groups/Cohorts HT-1 patients on Orfadin treatment
HT-1 patients on Orfadin (nitisinone) treatment
Intervention: Drug: Nitisinone
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: November 5, 2019)		 315
Original Estimated Enrollment
 (submitted: December 16, 2014)		 400
Actual Study Completion Date September 30, 2019
Actual Primary Completion Date September 30, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  • No exclusion criteria
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Austria,   Belgium,   Czechia,   Denmark,   Finland,   France,   Germany,   Hungary,   Ireland,   Italy,   Netherlands,   Norway,   Poland,   Portugal,   Spain,   Sweden,   United Kingdom
Removed Location Countries Croatia,   Czech Republic,   Greece,   Kosovo,   Lithuania,   Macedonia, The Former Yugoslav Republic of,   Romania,   Slovakia,   Switzerland
 
Administrative Information
NCT Number NCT02320084
Other Study ID Numbers Sobi.NTBC-005
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement
Plan to Share IPD: Undecided
Plan Description: Discussions ongoing with regards to data sharing/registry.
Current Responsible Party Swedish Orphan Biovitrum
Original Responsible Party Same as current
Current Study Sponsor Swedish Orphan Biovitrum
Original Study Sponsor Same as current
Collaborators Not Provided
Investigators
Study Director: Mattias Rudebeck Swedish Orphan Biovitrum
PRS Account Swedish Orphan Biovitrum
Verification Date January 2020