The primary purpose of the study is to investigate whether the new 20 mg capsule of Orfadin® (nitisinone) has a similar bioavailability in the body (gives the same concentration in the blood) as two of the marketed capsules of 10 mg. The secundary…
ID
Source
Brief title
Condition
- Metabolic and nutritional disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The area under the serum concentration vs. time profile during 72 hours after
dose (AUC72h).
The maximum serum concentration (Cmax).
Secondary outcome
AUC72h, AUC from time zero to infinity (AUC*), Cmax, time to reach Cmax (tmax),
terminal half-life (t*z), oral clearance (CL/F), and apparent volume of
distribution (Vz/F).
AEs, clinical chemistry, hematology, urinalysis.
Background summary
Orfadin® (nitisinone) is used in the treatment of hereditary tyrosinemia type 1
(HT-1), an inborn error of metabolism. (See Patient Information Leaflet (PIL),
for more detailed information.) Before the market authorization of Orfadin
(2002 in the US and 2005 in Europe) there was no available treatment for this
fatal condition and patients therefore died very young. Since some patients
have now reached adulthood, they need higher daily doses and use multiple
capsules of 10 mg nitisinone. The new 20-mg strength tested in this study is
easier to use.
Study objective
The primary purpose of the study is to investigate whether the new 20 mg
capsule of Orfadin® (nitisinone) has a similar bioavailability in the body
(gives the same concentration in the blood) as two of the marketed capsules of
10 mg.
The secundary purpose is to investigate the safety of the nitisinone capsules.
Study design
This is an open-label, randomized 2-way crossover study with 21-day washout
between doses.
Intervention
All volunteers will receive, in randomized order:
- 20 mg nitisinone as 1 capsule of 20 mg
- 20 mg nitisinone as 2 capsules of 10 mg
Study burden and risks
During the study a number of assessments will be performed that may be
considered more or less stressfull.
These are:
- Bloodsampling via venopunction and intravenous cannule
- ECG
- slit-lamp examination
Stationsweg 163
Zuidlaren 9471 GP
NL
Stationsweg 163
Zuidlaren 9471 GP
NL
Age
Inclusion criteria
healthy male and female subjects
18-55 yrs, inclusive
BMI: 18.5-30.0 kg/m2, inclusive
smoking <<= 10 cigarettes per day
Exclusion criteria
Suffering from hepatitis B, hepatitis C, cancer or HIV/AIDS. In case of participation in another drug study within 90 days before the start of this study or being a blood donor > 50 mL within 60 days from the start of the study. In case of donating more than 1.5 liters of blood in the 10 months prior the start of this study.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | 2012-005552-42 |
| CCMO | NL44365.056.13 |