Phase 1
Completed N=18
Taste and Palatability of Orfadin Suspension
Hereditary Tyrosinemia, Type I
Source: ClinicalTrials.gov NCT01734889 ↗
Enrolled (actual)
18
Serious AEs
0.0%
Results posted
Mar 2014
Primary outcomePrimary: The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years — 4.0 units on a scale
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years |
4.0 | — |
| PRIMARY The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years |
5.0 | — |
| SECONDARY The Palatability Scores on Day 1 (Subjects 5 - < 18 Years) |
4.0 | — |
| SECONDARY The Palatability Scores on Day 2 (Subjects 5 - < 18 Years) |
4.0 | — |
| SECONDARY The Palatability Scores on Day 3 (Subjects 5 - < 18 Years) |
4.0 | — |
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT01734889). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.