DEC-NET Serial number ES532 | | Published online | 06/04/2006 14.01.00 | Last updated | 24/04/2006 9.11.57 | | | Other protocol ID number | N0544122046 | Current trial status | Closed to recruitment of participants: follow-up continuing | Major Disease (ICD9 class) | LIPIDOSES | Experimental drug |
Agalsidase Alfa
Treatment regimen (dosage and duration) 0.2mg/kg fortnightly for six months |
Gender | Both | Age (range) | 2- 18 years | Eligibility criteria | Inclusion criteria | confirmed diagnosis by enzymology and/or mutation analysis in males and mutation analysis in females | Exclusion criteria | < 2 years; participating in any other clinical trial | Trial design/methodology | Phase | 3 | Kind of study | Efficacy Safety
| Design | Abierto | 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). |
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Principal investigator | Name | Guillem Pintos-Morell | Institution | University Hospital Germans Trias i Pujol | Postal address | Ctra del Canyet | City | Badalona | Country | SPAIN | Phone | +34 93 497 8928 | Fax | +34 93 497 8843 | E-mail | gpintos.germanstrias@gencat.net |
International lead principal investigator (for international trials) | Name | Dr Uma Ramaswami | Institution | Addenbrooke's NHS Trust | Postal address | Dept. of paediatric endocrinology, diabetes and metabolism, Box 181, Hills Road, CB2 2QQ | City | Cambridge | Country | UNITED KINGDOM | Phone | 01223-216878 | Fax | 01223 586794 | E-mail | |
Promoter | TKT 5S (Industry) |
Participating countries | UNITED KINGDOM | ITALY | GERMANY | SPAIN |
Participating centres | Hospital Universitario Infantil Virgen del Rocio (Sevilla) | Hospital Germans Trias I Pujol (Badalona) |
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