Taste and Palatability of Orfadin Suspension
This study has been completed.
Sponsor:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier:
NCT01734889
First received: November 22, 2012
Last updated: March 26, 2013
Last verified: March 2013
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Hereditary Tyrosinemia Type 1 (HT-1) |
Drug: Nitisinone |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin. |
Resource links provided by NLM:
Genetics Home Reference related topics:
argininosuccinic aciduria
citrullinemia
N-acetylglutamate synthase deficiency
ornithine translocase deficiency
succinic semialdehyde dehydrogenase deficiency
tyrosinemia
MedlinePlus related topics:
Taste and Smell Disorders
Drug Information available for:
Nitisinone
U.S. FDA Resources
Further study details as provided by Swedish Orphan Biovitrum:
Primary Outcome Measures:
- The taste score for the last dose of the suspension on Day 3 for subjects 5 - <18 years [ Time Frame: Day 0 to Day 3 ] [ Designated as safety issue: No ]
- The acceptability score for the last dose of the suspension on day 3 for subjects < 5 years [ Time Frame: Day 0 to Day 3 ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- The palatability scores on Day 1 (subjects 5 - < 18 years) [ Time Frame: Day 0 to Day 1 ] [ Designated as safety issue: No ]
- The palatability scores on Day 2 (subjects 5 - < 18 years) [ Time Frame: Day 0 to Day 2 ] [ Designated as safety issue: No ]
- The palatability scores on Day 3 (subjects 5 - < 18 years) [ Time Frame: Day 0 to Day 3 ] [ Designated as safety issue: No ]
| Enrollment: | 18 |
| Study Start Date: | October 2012 |
| Study Completion Date: | March 2013 |
| Primary Completion Date: | March 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Orfadin suspension
Drug: nitisinone, oral suspension
|
Drug: Nitisinone
Oral 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.
Eligibility| Ages Eligible for Study: | 1 Month to 17 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01734889
Locations
| France | |
| Hopital Necker | |
| Paris, France | |
| Germany | |
| Universitätsklinikum Düsseldorf | |
| Düsseldorf, Germany | |
| Universitätsklinikum Heidelberg | |
| Heidelberg, Germany | |
| Klinikum der Universität München | |
| München, Germany | |
| United Kingdom | |
| Birmingham Children's Hospital | |
| Birmingham, United Kingdom | |
| Evelina Children's Hospital, St Thomas' Hospital | |
| London, United Kingdom | |
| St Mary's Hospital | |
| Manchester, United Kingdom | |
Sponsors and Collaborators
Swedish Orphan Biovitrum
Investigators
| Study Director: | Maarten de Chateau, MD, PhD | Swedish Orphan Biovitrum |
More Information
No publications provided
| Responsible Party: | Swedish Orphan Biovitrum |
| ClinicalTrials.gov Identifier: | NCT01734889 History of Changes |
| Other Study ID Numbers: | Sobi.NTBC-002 |
| Study First Received: | November 22, 2012 |
| Last Updated: | March 26, 2013 |
| Health Authority: | United Kingdom: Medicines and Healthcare Products Regulatory Agency Germany: Federal Institute for Drugs and Medical Devices France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) |
Keywords provided by Swedish Orphan Biovitrum:
|
nitisinone Orfadin suspension taste acceptability |
Additional relevant MeSH terms:
|
Tyrosinemias Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Amino Acid Metabolism, Inborn Errors |
Metabolism, Inborn Errors Genetic Diseases, Inborn Metabolic Diseases Nitisinone Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 22, 2013