Hereditary Tyrosinemia, Type IPhase 1NCT01734889
Taste and Palatability of Orfadin Suspension
A Phase 1 study testing Nitisinone in people with Hereditary Tyrosinemia, Type I, run by Swedish Orphan Biovitrum at 7 sites in 3 countries.
Trial summary
The purpose of this study is to verify that pediatric patients, especially those who are not old enough to swallow capsules, accept the taste and palatability of a new suspension. Read more about this study
- Who is it for?
- Ages 1 month to 17 years
- Conditions the study is for
- Hereditary Tyrosinemia, Type I
- What is being tested?
- Nitisinone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 7 sitesin 3 countries
- Study dates
- Oct 2012to Mar 2013
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 1 month to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients with HT-1 currently managed on Orfadin (nitisinone) capsules.
- Age from 1 month to less than 18 years.
- Signed informed consent.
Who cannot join
- Any medical condition which in the opinion of the investigator makes the subject unsuitable for inclusion.
- Enrollment in another concurrent clinical study, or intake of an investigational medicinal product (IMP), within one month prior to inclusion in this study.
- Foreseeable inability to cooperate with given instructions or study procedures.
Trial locations and contacts
7 sites in 3 countries.
7 sites in 3 countries
Germany3 sites
Universitätsklinikum Düsseldorf
Düsseldorf
Universitätsklinikum Heidelberg
Heidelberg
Klinikum der Universität München
München
United Kingdom3 sites
Birmingham Children's Hospital
Birmingham
Evelina Children's Hospital, St Thomas' Hospital
London
St Mary's Hospital
Manchester
About this study
Official title: Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin.
The purpose of this study is to verify that pediatric patients, especially those who are not old enough to swallow capsules, accept the taste and palatability of a new suspension.
Read the detailed description
This is an open, non-randomized, non-controlled, multiple-dose study in 18 pediatric patients. The treatment period is three days, and during the study the subjects will rate the taste and palatability of the suspension or (for younger children) their parents will rate the child´s acceptance of the suspension.
The study consists of a screening period, a 3 day treatment period and a 1 week follow-up period.
- Conditions
- Hereditary Tyrosinemia, Type I
- Being tested
- Nitisinone
- Keywords
- nitisinone, Orfadin, suspension, taste, acceptability
- Medical subject headings
- Tyrosinemias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NitisinoneDrug
Oral suspension
| Study group | Role | What they receive |
|---|---|---|
| Orfadin suspension | Experimental | Drug: nitisinone, oral suspension Receives: Drug: Nitisinone |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years
Patients rated the taste of the suspension. The following grading was applied: 5 (very good taste), 4 (good taste), 3 (neither good nor bad taste), 2 (bad taste) and 1 (very bad taste).
Measured: Day 3
The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years
The parents of patients aged <5 years rated their child´s acceptability of the suspension. The following grading was applied: 5 (very well), 4 (well), 3 (neither well nor badly), 2 (badly) and 1 (very badly).
Measured: Day 3
Secondary outcomes (3)
The Palatability Scores on Day 1 (Subjects 5 - < 18 Years)
Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).
Measured: Day 1
The Palatability Scores on Day 2 (Subjects 5 - < 18 Years)
Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).
Measured: Day 2
The Palatability Scores on Day 3 (Subjects 5 - < 18 Years)
Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).
Measured: Day 3
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Nov 2012 (estimated)
- Study startOct 2012
- Primary completionMar 2013
- Study completionMar 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Erik Brouwer, MD, Study director, Swedish Orphan Biovitrum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01734889
- Sponsor's protocol ID
- Sobi.NTBC-002
- Status last verified
- Oct 2014
- First submitted
- 22 Nov 2012
- Last updated
- 6 Nov 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin.
- ClinicalTrials.gov ID
- NCT01734889
- Acronym / sponsor ID
- Sobi.NTBC-002
- Conditions
- Hereditary Tyrosinemia, Type I
- Keywords
- nitisinone, Orfadin, suspension, taste, acceptability
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- Oct 2012
- Primary completion
- Mar 2013 (actual)
- Study completion
- Mar 2013 (actual)
- First posted
- 28 Nov 2012 (estimated)
- Results
- Posted 4 Mar 2014
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 verify that pediatric patients, especially those who are not old enough to swallow capsules, accept the taste and palatability of a new suspension.
Detailed description
This is an open, non-randomized, non-controlled, multiple-dose study in 18 pediatric patients. The treatment period is three days, and during the study the subjects will rate the taste and palatability of the suspension or (for younger children) their parents will rate the child´s acceptance of the suspension.
The study consists of a screening period, a 3 day treatment period and a 1 week follow-up period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Orfadin suspension | Experimental | Drug: NitisinoneDrug: nitisinone, oral suspension |
Who may qualify
- Ages
- 1 month to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Patients with HT-1 currently managed on Orfadin (nitisinone) capsules.
- Age from 1 month to less than 18 years.
- Signed informed consent.
Key exclusion criteria (3 listed)
- Any medical condition which in the opinion of the investigator makes the subject unsuitable for inclusion.
- Enrollment in another concurrent clinical study, or intake of an investigational medicinal product (IMP), within one month prior to inclusion in this study.
- Foreseeable inability to cooperate with given instructions or study procedures.
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 |
|---|---|---|
| NitisinoneOral suspension | Drug | Orfadin suspension |
Primary outcome
The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years
Time frame: Day 3
Patients rated the taste of the suspension. The following grading was applied: 5 (very good taste), 4 (good taste), 3 (neither good nor bad taste), 2 (bad taste) and 1 (very bad taste).
The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years
Time frame: Day 3
The parents of patients aged <5 years rated their child´s acceptability of the suspension. The following grading was applied: 5 (very well), 4 (well), 3 (neither well nor badly), 2 (badly) and 1 (very badly).
3 secondary outcomes
The Palatability Scores on Day 1 (Subjects 5 - < 18 Years)
Time frame: Day 1
The Palatability Scores on Day 2 (Subjects 5 - < 18 Years)
Time frame: Day 2
The Palatability Scores on Day 3 (Subjects 5 - < 18 Years)
Time frame: Day 3
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Erik Brouwer, MD study director
Swedish Orphan Biovitrum
7 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Nitisinone in people with Hereditary Tyrosinemia, Type I, run by Swedish Orphan Biovitrum at 7 sites in 3 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 3 inclusion and 3 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 6 Nov 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Germany, United Kingdom, France. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Mar 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years (time frame: Day 3). The study lists 2 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 6 Nov 2014.