An open-label, non-randomized, sequential, multicenter study to evaluate the pharmacokinetics, efficacy and safety of once daily dosing compared to twice daily dosing of Orfadin in patients diagnosed with hereditary tyrosinemia type 1
- Conditions
- hereditary tyrosinemia type 1MedDRA version: 17.1Level: LLTClassification code 10069462Term: Tyrosinemia type ISystem Organ Class: 100000004850Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
- Registration Number
- EUCTR2013-004132-29-SE
- Lead Sponsor
- Swedish Orphan Biovitrum AB (Publ)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 20
1. Male and female patients of all ages diagnosed with HT-1.
2. Patients currently well-controlled, as judged by the investigator, on twice daily (or more frequent) dosing with Orfadin.
3. Stable lab values, including liver values <2 ULN (ALP, ALT, AST, bilirubin, INR).
4. Women of childbearing potential willing to use adequate contraception
5. Signed informed consent/assent.
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0
1. Patients who have been previously treated with once daily Orfadin, even if later converted to twice daily dosing.
2. Any medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.
3. Enrollment in another concurrent clinical interventional study within three months prior to inclusion in this study.
4. Pregnant women.
5. Lactating women.
6. Previous liver transplantation
7. Patients who have recently (past 4 weeks prior to inclusion) started any new medication for a previously undiagnosed illness/disease.
8. Known hepatitis B, hepatitis C or HIV infection.
9. Foreseeable inability to cooperate with given instructions or study procedures.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the steady-state exposure to nitisinone during once and twice daily dosing of Orfadin;Secondary Objective: - To evaluate the efficacy of Orfadin during once daily dosing.<br>- To evaluate the safety of Orfadin during once and twice daily dosing.;Primary end point(s): Minimum (Cmin) serum concentrations of nitisinone after at least 4 weeks of treatment on each dosage regimen. Cmin = concentration in the sample taken immediately before dosing.;Timepoint(s) of evaluation of this end point: D1, after 4w + 10 days on 2x daily dosing with IMP - predose, after 4w + 10 days on 1x daily dosing with IMP - predose<br>
- Secondary Outcome Measures
Name Time Method