Dettagli per singolo studio

DEC-NET Serial number GB446

Published online	18/07/2005 10.30.00
Last updated	18/07/2005 12.39.11


This trial has been approved by an ethics committee
Current trial status	Closed to recruitment of participants: follow-up continuing
First subject enrolment Target N. of subjects	01/2003 40
Major Disease (ICD9 class)	CEREBRAL DEGENERATION OF CHILDHOOD IN OTHER DISEAS
Experimental drug
Agalsidase Alfa Treatment regimen (dosage and duration) 0.2mg/kg fortnightly for six months
Gender	Both
Age (range)	less than 18
Eligibility criteria
Inclusion criteria
Confirmed diagnosis by enzymology and/or mutation analysis in males and mutation analysis in females
Exclusion criteria
Less than 2 years; participating in any other clinical trial
Trial design/methodology
Phase	3
Kind of study	Efficacy Safety
Design
Purpose of study
The objectives of this clinical trial are to evaluate the safety (primary objective) of multiple intravenous (IV) doses of Replagal every other week over a 24-week period in up to 40 children (European multicentre) aged < 18 years old with Fabry Disease. Safety will be determined by standard clinical and laboratory measurements. These patients will be entered into the Fabry Outcome Survey (FOS) in order to capture long-term (5 years or more) outcome data on enzyme replacement therapy with Replagal.
Primary outcomes
Safety of replagal in children between 2 -18 years
Secondary outcomes
Efficacy
Summary of study design, objectives, and ongoing research findings
This is an open label study. Prior to enrolment, patients will be screened by medical history to ensure that they meet study eligibility criteria for the diagnosis of Fabry disease. Prior to beginning dosing with Replagal, a number of baseline studies will be performed. Patients will then receive multiple infusions of Replagal at a dose of 0.2 mg/kg/dose every other week for a total of 24 weeks. The primary endpoint of this study will be an assessment of safety. In addition, a number of baseline measurements will form the basis for the assessment of the long-term outcome in FOS. At the end of the study patients will be offered to continue follow up in the Fabry Outcome Survey (FOS).