Dosing Study of Replagal in Patients With Fabry Disease


Phase 2 Results

Eligibility Criteria

Patients with Fabry disease participating in 00-N-0185/TKT011 or 02-N-0220/TKT015 may be eligible. No other Fabry patients will be eligible.
Patients losing GFR at a rate greater than 5 ml/min/year despite ERT with agalsidase alfa for greater than or equal to 2.5 years in 00-N-0185/TKT/003/006/011 Study or ERT over greater than or equal to 1.0 years in 02-N-0220/TKT/010/015 Study.
Patients who at least twice demonstrated significant improvement or normalization of sweat function (by QSART or thermoregulatory sweat test) or reduction in serum creatinine by at least 10% but return to the pre-infusion state before the subsequent biweekly enzyme infusion.
Patients who freely agree to participate in this study and understand the nature, risks and benefits of this study and give their written informed consent.
Patients with Fabry disease, who are not already part of 00-N-0185/TKT011 or 02-N-0220/TKT015.
Patients on these protocols who have stable serum creatinine (or a lesser rise in serum creatinine than stipulated in the inclusion criteria), and do not show other objective evidence of incomplete clinical response between biweekly infusions (e.g. sweat function).
Patients who have begun dialysis or who have received a renal transplant.
Patients who cannot tolerate the study procedures or who are unable or unwilling to travel to the study center as required by this protocol.
Patients with an intercurrent medical condition that would render them unsuitable for mthe study e.g. HIV, diabetes. The reason is that the pathologies of these conditions will be significant confounders in assessing the effect of the experimental therapy and its adverse events.
Patients who in the opinion of the investigator (for whatever reason) are thought to be unsuitable for the study.