Hereditary tyrosinemia type 1 (HT-1) MedDRA version: 14.1 Level: LLT Classification code 10069459 Term: Tyrosinaemia type I System Organ Class: 100000004850
Conditions
Interventions
Product Name: Orfadin oral suspension 4mg/ml
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Nitisinone
CAS Number: 104206-65-7
Concentration unit: mg/ml milligram(s)/millilitre
Concentratio
Sponsors
Swedish Orphan Biovitrum AB
Eligibility
Sex/Gender
All
Inclusion 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. Are the trial subjects under 18? yes Number of subjects for this age range: 18 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 |
|---|---|
| Main Objective: To determine the acceptability of the suspension in the pediatric population.;Secondary Objective: To assess the taste of the suspension on Days 1 and 2 for subjects 5- < 18 years. To assess the acceptability of the suspension on Days 1 and 2 for subjects < 5years. To assess the palatability of the suspension on Days 1, 2 and 3 for subjects 5- < 18 years. To assess the overall acceptability of the suspension in subjects swallowing the capsules whole and subjects mixing capsule contents with food, respectively. To assess the tolerability of the suspension.;Primary end point(s): The taste score for the last dose of the suspension on Day 3 for subjects 5 -< 18 years. The acceptability score for the last dose of the suspension on Day 3 for subjects < 5 years.;Timepoint(s) of evaluation of this end point: Day 3 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Taste scores on Days 1 and 2 (subjects 5-<18 years). The acceptability scores on Days 1 and 2 (subjects < 5 years). The palatability scores on Days 1, 2 and 3 (subjects 5-<18 years). The overall acceptability response (subjects 0-<18 years). Adverse events.;Timepoint(s) of evaluation of this end point: Days 1, 2 and 3. | — |
Countries
Germany, United Kingdom
Contacts
Public ContactMedical Director
Swedish Orphan Biovitrum AB
Outcome results
None listed