NCT01734889

Brief 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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Oct 2012

Shorter than P25 for phase_1

Geographic Reach
3 countries

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2012

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

November 22, 2012

Completed
6 days until next milestone

First Posted

Study publicly available on registry

November 28, 2012

Completed
3 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2013

Completed
1 year until next milestone

Results Posted

Study results publicly available

March 4, 2014

Completed
Last Updated

November 6, 2014

Status Verified

October 1, 2014

Enrollment Period

5 months

First QC Date

November 22, 2012

Results QC Date

January 21, 2014

Last Update Submit

October 31, 2014

Conditions

Hereditary Tyrosinemia, Type I

Keywords

nitisinoneOrfadinsuspensiontasteacceptability

Outcome Measures

Primary Outcomes (2)

  • The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years

    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).

    Day 3

  • The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years

    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).

    Day 3

Secondary Outcomes (3)

  • The Palatability Scores on Day 1 (Subjects 5 - < 18 Years)

    Day 1

  • The Palatability Scores on Day 2 (Subjects 5 - < 18 Years)

    Day 2

  • The Palatability Scores on Day 3 (Subjects 5 - < 18 Years)

    Day 3

Study Arms (1)

Orfadin suspension

EXPERIMENTAL

Drug: nitisinone, oral suspension

Drug: Nitisinone

Interventions

NitisinoneDRUG

Oral suspension

Orfadin suspension

Eligibility Criteria

Age1 Month - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Patients with HT-1 currently managed on Orfadin (nitisinone) capsules.
  • Age from 1 month to less than 18 years.
  • Signed informed consent.

You may not qualify if:

  • Foreseeable inability to cooperate with given instructions or study procedures.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Hopital Necker

Paris, France

Location

Universitätsklinikum Düsseldorf

Düsseldorf, Germany

Location

Universitätsklinikum Heidelberg

Heidelberg, Germany

Location

Klinikum der Universität München

München, Germany

Location

Birmingham Children's Hospital

Birmingham, United Kingdom

Location

Evelina Children's Hospital, St Thomas' Hospital

London, United Kingdom

Location

St Mary's Hospital

Manchester, United Kingdom

Location

MeSH Terms

Conditions

Tyrosinemias

Interventions

nitisinone

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Results Point of Contact

Title
Medical Director
Organization
Swedish Orphan Biovitrum
erik.brouwer@sobi.com
+46 8 697 20 00

Study Officials

  • Erik Brouwer, MD

    Swedish Orphan Biovitrum

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 22, 2012

First Posted

November 28, 2012

Study Start

October 1, 2012

Primary Completion

March 1, 2013

Study Completion

March 1, 2013

Last Updated

November 6, 2014

Results First Posted

March 4, 2014

Record last verified: 2014-10

Locations

FR(1)GB(3)DE(3)