Hereditary Tyrosinemia, Type IOPALNCT02320084
Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
An observational study of Hereditary Tyrosinemia, Type I, run by Swedish Orphan Biovitrum at 77 sites in 17 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hereditary Tyrosinemia, Type I
- What is being tested?
- Nitisinone
- How many people
- 315 participants
- Where
- 77 sitesin 17 countries
- Study dates
- Sep 2013to 30 Sep 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All patients receiving Orfadin is eligible for the study
Who may be able to join
- All HT-1 patients receiving Orfadin treatment are eligible for entry.
Who cannot join
- No exclusion criteria
Trial locations and contacts
77 sites in 17 countries.
77 sites in 17 countries
Spain15 sites
Swedish Orphan Biovitrum Investigational Site
Albacete
Swedish Orphan Biovitrum Investigational Site
Almería
Swedish Orphan Biovitrum Investigational Site
Badalona
Swedish Orphan Biovitrum Investigational Site
Barakaldo
Swedish Orphan Biovitrum Investigational Site
Barcelona
Swedish Orphan Biovitrum Investigational Site
Las Palmas de Gran Canaria
Swedish Oprhan Biovitrum Investigational Site
Madrid
Swedish Orphan Biovitrum Investigational Site
Madrid
Swedish Orphan Biovitrum Investigational Site
Málaga
Swedish Orphan Biovitrum Investigational Site
Mérida
Swedish Orphan Biovitrum Investigational Site
Murcia
Swedish Orphan Biovitrum Investigational Site
Pamplona
Swedish Orphan Biovitrum Investigational Site
Santiago
Swedish Orphan Biovitrum Investigational Site
Seville
Swedish Orphan Biovitrum Investigational Site
Valencia
Italy10 sites
Swedish Orphan Biovitrum Investigational Site
Bari
Swedish Orphan Biovitrum Investigational Site
Catania
Swedish Orphan Biovitrum Investigational Site
Florence
Swedish Orphan Biovitrum Investigational Site
Milan
Swedish Orphan Biovitrum Investigational Site
Monza
Swedish Orphan Biovitrum Investigational Site
Naples
Swedish Orphan Biovitrum Investigational Site
Padova
Swedish Orphan Biovitrum Investigational Site
Roma
Swedish Orphan Biovitrum Investigational Site
Saliceto
Swedish Orphan Biovitrum Investigational Site
Torino
Germany9 sites
Swedish Orphan Biovitrum Investigational Site
Düsseldorf
Swedish Orphan Biovitrum Investigational Site
Erlangen
Swedish Orphan Biovitrum Investigational Site
Freiburg im Breisgau
Swedish Orphan Biovitrum Investigational Site
Hanover
Swedish Orphan Biovitrum Investigational Site
Heidelberg
Swedish Orphan Biovitrum Investigational Site
Leipzig
Swedish Orphan Biovitrum Investigational Site
Magdeburg
Swedish Orphan Biovitrum Investigational Site
München
Swedish Orphan Biovitrum Investigational Site
Reutlingen
France7 sites
Swedish Orphan Biovitrum Investigational Site
Angers
Swedish Oprhan Biovitrum Investigational Site
Lille
Swedish Orphan Biovitrum Investigational Site
Lyon
Swedish Orphan Biovitrum Investigational Site
Nantes
Swedish Oprhan Biovitrum Investigational Site
Paris
Swedish Orphan Biovitrum Investigational Site
Paris
Swedish Orphan Biovitrum Investigational Site
Talence
United Kingdom7 sites
Swedish Oprhan Biovitrum Investigational Site
Birmingham
Swedish Orphan Biovitrum Investigational Site
Bradford
Swedish Oprhan Biovitrum Investigational Site
London
Swedish Orphan Biovitrum Investigational Site
London
Swedish Oprhan Biovitrum Investigational Site
Manchester
Swedish Orphan Biovitrum Investigational Site
Manchester
Swedish Orphan Biovitrum Investigational Site
Sheffield
Belgium5 sites
Swedish Orphan Biovitrum Investigational Site
Antwerp
Swedish Orphan Biovitrum Investigational Site
Brussels
Swedish Orphan Biovitrum Investigational Site
Leuven
Swedish Orphan Biovitrum Investigational Site
Montegnée
Swedish Orphan Biovitrum Investigational Site
Woluwe-Saint-Lambert
Norway5 sites
Swedish Orphan Biovitrum Investigational Site
Fredrikstad
Swedish Orphan Biovitrum Investigational Site
Levanger
Swedish Oprhan Biovitrum Investigational Site
Oslo
Swedish Orphan Biovitrum Investigational Site
Oslo
Swedish Orphan Biovitrum Investigational Site
Tromsø
Sweden5 sites
Swedish Orphan Biovitrum Investigational Site
Gothenburg
Swedish Orphan Biovitrum Investigational Site
Stockholm, 14186
Swedish Orphan Biovitrum Investigational Site
Stockholm
Swedish Orphan Biovitrum Investigational Site
Umeå
Swedish Orphan Biovitrum Investigational Site
Uppsala
Austria2 sites
Swedish Orphan Biovitrum Investigational Site
Innsbruck
Swedish Orphan Biovitrum Investigational Site
Vienna
Finland2 sites
Swedish Orphan Biovitrum Investigational Site
Tampere
Swedish Orphan Biovitrum Investigational Site
Turku
Hungary2 sites
Swedish Orphan Biovitrum Investigational Site
Budapest
Swedish Oprhan Biovitrum Investigational Site
Szeged
Netherlands2 sites
Swedish Oprhan Biovitrum Investigational Site
Amsterdam
Swedish Orphan Biovitrum Investigational Site
Utrecht
Poland2 sites
Swedish Oprhan Biovitrum Investigational Site
Warsaw
Swedish Orphan Biovitrum Investigational Site
Warsaw
About this study
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
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.
Read the 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.
- Conditions
- Hereditary Tyrosinemia, Type I
- Being tested
- Nitisinone
- Keywords
- HT-1, Hereditary Tyrosinemia, Type I, hypertyrosinemia type 1, Orfadin, Long term safety, Hereditary Tyrosinemia Type 1
- Medical subject headings
- Tyrosinemias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NitisinoneDrug
Also called Orfadin
Nitisinone according to prescription
| Study group | Role | What they receive |
|---|---|---|
| HT-1 patients on Orfadin treatment | HT-1 patients on Orfadin (nitisinone) treatment Receives: Drug: Nitisinone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of Adverse events related to hepatic function
Occurrence of Adverse events related to hepatic function
Measured: from 2005
Occurrence of Adverse events related to renal function
Occurrence of Adverse events related to renal function
Measured: from 2005
Occurrence of Adverse events related to Ophthalmological function
Occurrence of Adverse events related to Ophthalmological function
Measured: from 2005
Occurrence of Adverse events related to hematological function
Occurrence of Adverse events related to hematological function
Measured: from 2005
Occurrence of Adverse events related to cognitive developmental function
Occurrence of Adverse events related to cognitive developmental function
Measured: from 2005
Secondary outcomes (4)
Occurrence of death
Occurrence of death
Measured: from 2005
Occurrence of liver transplantation
Occurrence of liver transplantation
Measured: from 2005
Occurrence of other Adverse Events
Occurrence of Adverse Events other than those related to hepatic, renal, ophthalmic, hematological or cognitive functions
Measured: from 2005
Occurrence of discontinuation of Orfadin treatment
Occurrence of discontinuation of Orfadin treatment
Measured: from 2005
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2014 (estimated)
- Study startSep 2013
- Primary completion30 Sep 2019
- Study completion30 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Tyrosinemia, Type IStudies of the same condition, open and under way.
- More trials run by Swedish Orphan BiovitrumThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 315 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Physician, Study director, Swedish Orphan Biovitrum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02320084
- Sponsor's protocol ID
- Sobi.NTBC-005
- Acronym
- OPAL
- Status last verified
- Sep 2024
- First submitted
- 23 Oct 2014
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided: Discussions ongoing with regards to data sharing/registry.
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02320084
- Acronym / sponsor ID
- OPAL / Sobi.NTBC-005
- Conditions
- Hereditary Tyrosinemia, Type I
- Keywords
- HT-1, Hereditary Tyrosinemia, Type I, hypertyrosinemia type 1, Orfadin, Long term safety, Hereditary Tyrosinemia Type 1
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 315 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- Sep 2013 (actual)
- Primary completion
- 30 Sep 2019 (actual)
- Study completion
- 30 Sep 2019 (actual)
- First posted
- 19 Dec 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HT-1 patients on Orfadin treatment | – | Drug: NitisinoneHT-1 patients on Orfadin (nitisinone) treatment |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All patients receiving Orfadin is eligible for the study
Key inclusion criteria (1 listed)
- All HT-1 patients receiving Orfadin treatment are eligible for entry.
Key exclusion criteria (1 listed)
- No exclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NitisinoneAlso: OrfadinNitisinone according to prescription | Drug | HT-1 patients on Orfadin treatment |
Primary outcome
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
4 secondary outcomes
Occurrence of death
Time frame: from 2005
Occurrence of liver transplantation
Time frame: from 2005
Occurrence of other Adverse Events
Time frame: from 2005
Occurrence of discontinuation of Orfadin treatment
Time frame: from 2005
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Individual participant dataSharing planned: Undecided. Discussions ongoing with regards to data sharing/registry.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Physician study director
Swedish Orphan Biovitrum
77 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Tyrosinemia, Type I, run by Swedish Orphan Biovitrum at 77 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 77 sites in Spain, Italy, Germany, France, United Kingdom and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Occurrence of Adverse events related to hepatic function (time frame: from 2005). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.