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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Orfadin suspension | Experimental | Drug: nitisinone, oral suspension |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Nitisinone | Drug | Oral suspension |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 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). | Day 3 |
| Measure | Description | Time Frame |
|---|---|---|
| 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). | Day 1 |
| The Palatability Scores on Day 2 (Subjects 5 - < 18 Years) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Erik Brouwer, MD | Swedish Orphan Biovitrum | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hopital Necker | Paris | France | ||||
| Universitätsklinikum Düsseldorf |
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The study recruitment was performed at seven medical clinics in three countries (United Kingdom, Germany and France). First subject was enrolled on 09 November 2012 and last subject´s last visit was on 01 March 2013.
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| ID | Title | Description |
|---|---|---|
| FG000 | Orfadin Suspension | Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Orfadin Suspension | Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | Full analysis set: All subjects who received at least one dose of study drug and had at least one taste or acceptability assessments | Posted | Median | Full Range | units on a scale | Day 3 |
|
|
Adverse events were recorded upon administration of first dose until last study visit 1 week after last dose. Serious Adverse Events were recorded from subject signed informed consent until 28 days after last dose.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Orfadin Suspension | Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Swedish Orphan Biovitrum | +46 8 697 20 00 | erik.brouwer@sobi.com |
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| ID | Term |
|---|---|
| D020176 | Tyrosinemias |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C077073 | nitisinone |
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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). |
| 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). | Day 3 |
| Düsseldorf |
| Germany |
| Universitätsklinikum Heidelberg | Heidelberg | Germany |
| Klinikum der Universität München | München | Germany |
| Birmingham Children's Hospital | Birmingham | United Kingdom |
| Evelina Children's Hospital, St Thomas' Hospital | London | United Kingdom |
| St Mary's Hospital | Manchester | United Kingdom |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Counts |
|---|
| Participants |
|
|
| Primary | 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). | Full analysis set: All subjects who received at least one dose of study drug and had at least one taste or acceptability assessments | Posted | Median | Full Range | units on a scale | Day 3 |
|
|
|
| Secondary | 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). | Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment. | Posted | Median | Full Range | units on a scale | Day 1 |
|
|
|
| Secondary | 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). | Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment. | Posted | Median | Full Range | units on a scale | Day 2 |
|
|
|
| Secondary | 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). | Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment. | Posted | Median | Full Range | units on a scale | Day 3 |
|
|
|
| 0 |
| 18 |
| 4 |
| 18 |
| Regurgitation | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 15.1 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
The results are intended to be published as a whole. If no comprehensive publication appears within 12 months of the trial termination, Investigators have the right to publish their results. Such data to be submitted to the Sponsor for review at least 60 days prior to submission for publication, public dissemination, or review by a publication committee. The Sponsor shall be entitled to make a reasoned request that publication be delayed for a period of up to 6 months.
| D009422 | Nervous System Diseases |
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |