Hereditary Tyrosinemia, Type I
Conditions
Keywords
nitisinone, Orfadin, suspension, taste, acceptability
Brief 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.
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.
Interventions
DRUGNitisinone
Oral suspension
Sponsors
Swedish Orphan Biovitrum
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Months to 17 Years
Healthy volunteers
No
Inclusion criteria
* Patients with HT-1 currently managed on Orfadin (nitisinone) capsules. * Age from 1 month to less than 18 years. * Signed informed consent.
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years | 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 | 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). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The Palatability Scores on Day 1 (Subjects 5 - < 18 Years) | Day 1 | 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). |
| The Palatability Scores on Day 2 (Subjects 5 - < 18 Years) | Day 2 | 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). |
| The Palatability Scores on Day 3 (Subjects 5 - < 18 Years) | Day 3 | 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). |
Countries
France, Germany, United Kingdom
Participant flow
Recruitment details
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.
Participants by arm
| Arm | Count |
|---|---|
| Orfadin Suspension Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening | 18 |
| Total | 18 |
Baseline characteristics
| Characteristic | Orfadin Suspension |
|---|---|
| Age, Categorical <=18 years | 18 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 8.03 years STANDARD_DEVIATION 5.6 |
| Region of Enrollment France | 2 participants |
| Region of Enrollment Germany | 4 participants |
| Region of Enrollment United Kingdom | 12 participants |
| Sex: Female, Male Female | 9 Participants |
| Sex: Female, Male Male | 9 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 4 / 18 |
| serious Total, serious adverse events | 0 / 18 |
Outcome results
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).
Time frame: Day 3
Population: Full analysis set: All subjects who received at least one dose of study drug and had at least one taste or acceptability assessments
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Age 5-<18 Years | The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years | 5.0 units on a scale |
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).
Time frame: Day 3
Population: Full analysis set: All subjects who received at least one dose of study drug and had at least one taste or acceptability assessments
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Age 5-<18 Years | The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years | 4.0 units on a scale |
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).
Time frame: Day 1
Population: Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Age 5-<18 Years | The Palatability Scores on Day 1 (Subjects 5 - < 18 Years) | 4.0 units on a scale |
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).
Time frame: Day 2
Population: Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Age 5-<18 Years | The Palatability Scores on Day 2 (Subjects 5 - < 18 Years) | 4.0 units on a scale |
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).
Time frame: Day 3
Population: Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Age 5-<18 Years | The Palatability Scores on Day 3 (Subjects 5 - < 18 Years) | 4.0 units on a scale |