Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1

• Status: Completed
• Trial end date: 2015-09-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to look at the steady-state serum concentrations of nitisinone when switching from twice daily and once daily dosing.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Swedish Orphan Biovitrum
• Treatments: Nitisinone

Criteria

Inclusion Criteria:

• Male and female patients of all ages diagnosed with HT-1.

• Patients currently well-controlled, as judged by the investigator, on twice daily (or more frequent) dosing with Orfadin.

• Stable lab values, including liver values <2 ULN (ALP, ALT, AST, bilirubin, INR).

• Women of childbearing potential willing to use adequate contraception

• Signed informed consent/assent.

Exclusion Criteria:

• Patients who have been previously treated with once daily Orfadin, even if later converted to twice daily dosing.

• Any medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.

• Enrollment in another concurrent clinical interventional study within three months prior to inclusion in this study.

• Pregnant women.

• Lactating women.

• Previous liver transplantation.

• Patients who have recently (past 4 weeks prior to inclusion) started any new medication for a previously undiagnosed illness/disease.

• Known hepatitis B, hepatitis C or HIV infection.

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