Trial Outcomes & Findings for Taste and Palatability of Orfadin Suspension (NCT NCT01734889)

Results Overview

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).

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

18 participants

Primary outcome timeframe

Day 3

Results posted on

2014-11-06

Participant Flow

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.

Participant milestones

Participant milestones
Measure	Orfadin Suspension Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
Overall Study STARTED	18
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Taste and Palatability of Orfadin Suspension

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Orfadin Suspension n=18 Participants Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
Age, Categorical <=18 years	18 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	8.03 years STANDARD_DEVIATION 5.60 • n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Region of Enrollment France	2 participants n=5 Participants
Region of Enrollment Germany	4 participants n=5 Participants
Region of Enrollment United Kingdom	12 participants n=5 Participants

PRIMARY outcome

Timeframe: 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

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).

Outcome measures

Outcome measures
Measure	Age 5-<18 Years n=12 Participants Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years	4.0 units on a scale Interval 2.0 to 5.0

PRIMARY outcome

Timeframe: 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

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).

Outcome measures

Outcome measures
Measure	Age 5-<18 Years n=6 Participants Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years	5.0 units on a scale Interval 4.0 to 5.0

SECONDARY outcome

Timeframe: 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.

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).

Outcome measures

Outcome measures
Measure	Age 5-<18 Years n=12 Participants Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
The Palatability Scores on Day 1 (Subjects 5 - < 18 Years)	4.0 units on a scale Interval 3.0 to 5.0

SECONDARY outcome

Timeframe: 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.

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).

Outcome measures

Outcome measures
Measure	Age 5-<18 Years n=12 Participants Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
The Palatability Scores on Day 2 (Subjects 5 - < 18 Years)	4.0 units on a scale Interval 2.0 to 5.0

SECONDARY outcome

Timeframe: 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.

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).

Outcome measures

Outcome measures
Measure	Age 5-<18 Years n=12 Participants Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
The Palatability Scores on Day 3 (Subjects 5 - < 18 Years)	4.0 units on a scale Interval 2.0 to 5.0

Adverse Events

Orfadin Suspension

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Orfadin Suspension n=18 participants at risk Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
Infections and infestations Nasopharyngitis	5.6% 1/18 • Number of events 1 • 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.
Gastrointestinal disorders Regurgitation	5.6% 1/18 • Number of events 1 • 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.
General disorders Pyrexia	5.6% 1/18 • Number of events 1 • 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.
Gastrointestinal disorders Diarrhoea	5.6% 1/18 • Number of events 1 • 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.
Gastrointestinal disorders Mouth haemorrhage	5.6% 1/18 • Number of events 1 • 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.

Additional Information

Medical Director

Swedish Orphan Biovitrum

Phone: +46 8 697 20 00

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER