The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of enzyme replacement therapy with recombinant human alpha-L-iduronidase \[Aldurazyme® (laronidase)\] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy measurements will also be evaluated in this study.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
100 U/kg every week
200 U/kg every week (Week 26 onwards)
Hôpital E. Herriot
Lyon, France
Johannes Gutenberg Universität
Kinderklinik, Mainz, Germany
Sophia Children's Hospital
Rotterdam, Netherlands
Willink Biochemical Genetics Unit Royal Hospital for Children
Manchester, United Kingdom
Safety Evaluation
Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.
Time frame: 52 weeks
Pharmacokinetics - Area Under the (Plasma Concentration-time) Curve (AUC∞)
AUC∞ is a measure of the total exposure to a drug.
Time frame: 52 weeks
Pharmacokinetics - Elimination Half Life (t1/2)
Half-life is the time it takes for the concentration of drug in plasma to decline by 50%.
Time frame: 52 weeks
Pharmacokinetics - Total Plasma Clearance (CL)
CL is volume of the body fluid cleared of the drug per unit of time.
Time frame: 52 weeks
Pharmacokinetics - Volume of Distribution (Vz)
Vz is the volume that relates the amount of drug in the body after absorption is complete to the concentration of drug in the plasma.
Time frame: 52 weeks
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