A Phase 4 interventional study of recombinant alpha-galactosidase A in Fabry Disease, sponsored by Assistance Publique - Hôpitaux de Paris. Status unknown at 1 site in France. Open to female participants aged 15 Years and older. Per ClinicalTrials.gov, last updated 2007-06-18.
Sponsored by Assistance Publique - Hôpitaux de Paris · Phase 4, Interventional, and Treatment
Fabry disease (OMIM 301500) is an X-linked inborn error of sphingolipid metabolism resulting from the deficiency of the lysosomal enzyme alpha-galactosidase A. Heterozygous females for Fabry disease may be symptomatic with cardiac, renal or cerebrovascular involvement. Clearance of Gb3 and stabilization of renal function has been demonstrated in male patients treated with agalsidase beta (FABRAZYME). In contrast, no randomized, controlled study of the efficacy of recombinant alpha-galactosidase A has been reported in heterozygotes for Fabry disease.
The primary objective is to evaluate cardiac left ventricular mass (measured with echocardiography by unique investigator) in females over 15 years of age affected with Fabry disease receiving 70 mg of agalsidase beta every other week, as compared with an untreated controlled group matched for gender and age.
The secondary objectives include evaluation of :
Evaluation of tolerance and safety with :
242 studies on the registry are indexed under Fabry Disease; 54 are open to participants now.
This study's planned enrollment of 34 is above the median of 22 across 105 interventional studies indexed under Fabry Disease.
Browse Fabry Disease studies →Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,505 studies on the registry; 1,006 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Left ventricular mass
Time frame: 2 years
Posterior wall thickness, interventricular thickness, ECG, creatinaemia, urinary protein / creatinine ratio, microalbuminuria, urinary Gb3 level
Time frame: 2 years
This study is status unknown, as verified in Jun 2007. You cannot join it, but the record below documents what was studied.
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Assistance Publique - Hôpitaux de Paris