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Taste and Palatability of Orfadin Suspension

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ClinicalTrials.gov Identifier: NCT01734889
Recruitment Status : Completed
First Posted : November 28, 2012
Results First Posted : March 4, 2014
Last Update Posted : November 6, 2014
Sponsor:
Information provided by (Responsible Party):
Swedish Orphan Biovitrum

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hereditary Tyrosinemia, Type I
Intervention Drug: Nitisinone
Enrollment 18
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.
Pre-assignment Details  
Arm/Group Title Orfadin Suspension
Hide Arm/Group Description Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
Period Title: Overall Study
Started 18
Completed 18
Not Completed 0
Arm/Group Title Orfadin Suspension
Hide Arm/Group Description Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
Overall Number of Baseline Participants 18
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
<=18 years
18
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 18 participants
8.03  (5.60)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
Female
9
  50.0%
Male
9
  50.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 18 participants
France 2
Germany 4
United Kingdom 12
1.Primary Outcome
Title The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set: All subjects who received at least one dose of study drug and had at least one taste or acceptability assessments
Arm/Group Title Age 5-<18 Years
Hide Arm/Group Description:
Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
Overall Number of Participants Analyzed 12
Median (Full Range)
Unit of Measure: units on a scale
4.0
(2.0 to 5.0)
2.Primary Outcome
Title The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set: All subjects who received at least one dose of study drug and had at least one taste or acceptability assessments
Arm/Group Title Age <5 Years
Hide Arm/Group Description:
Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
Overall Number of Participants Analyzed 6
Median (Full Range)
Unit of Measure: units on a scale
5.0
(4.0 to 5.0)
3.Secondary Outcome
Title The Palatability Scores on Day 1 (Subjects 5 - < 18 Years)
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.
Arm/Group Title Age 5-<18 Years
Hide Arm/Group Description:
Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
Overall Number of Participants Analyzed 12
Median (Full Range)
Unit of Measure: units on a scale
4.0
(3.0 to 5.0)
4.Secondary Outcome
Title The Palatability Scores on Day 2 (Subjects 5 - < 18 Years)
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.
Arm/Group Title Age 5-<18 Years
Hide Arm/Group Description:
Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
Overall Number of Participants Analyzed 12
Median (Full Range)
Unit of Measure: units on a scale
4.0
(2.0 to 5.0)
5.Secondary Outcome
Title The Palatability Scores on Day 3 (Subjects 5 - < 18 Years)
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.
Arm/Group Title Age 5-<18 Years
Hide Arm/Group Description:
Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
Overall Number of Participants Analyzed 12
Median (Full Range)
Unit of Measure: units on a scale
4.0
(2.0 to 5.0)
Time Frame 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.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Orfadin Suspension
Hide Arm/Group Description Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening
All-Cause Mortality
Orfadin Suspension
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Orfadin Suspension
Affected / at Risk (%) # Events
Total   0/18 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Orfadin Suspension
Affected / at Risk (%) # Events
Total   4/18 (22.22%)    
Gastrointestinal disorders   
Regurgitation  1  1/18 (5.56%)  1
Diarrhoea  1  1/18 (5.56%)  1
Mouth haemorrhage  1  1/18 (5.56%)  1
General disorders   
Pyrexia  1  1/18 (5.56%)  1
Infections and infestations   
Nasopharyngitis  1  1/18 (5.56%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.1
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Director
Organization: Swedish Orphan Biovitrum
Phone: +46 8 697 20 00
EMail: erik.brouwer@sobi.com
Layout table for additonal information
Responsible Party: Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier: NCT01734889     History of Changes
Other Study ID Numbers: Sobi.NTBC-002
First Submitted: November 22, 2012
First Posted: November 28, 2012
Results First Submitted: January 21, 2014
Results First Posted: March 4, 2014
Last Update Posted: November 6, 2014