Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease

• Status: Unknown status
• Trial end date: 2009-06-01
• Target enrollment: 0
• Participant gender: Female

Summary

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Criteria

Inclusion Criteria:

• Female patients over 15 years with clinical and biological evidence of Fabry disease (GLA gene mutation detected)

Exclusion Criteria:

• Pregnancy

• Allergy to agalsidase beta

• Congestive heart failure

• Creatinaemia > 135 µmol/l

• Medical history of stroke during the last year

• Medical history of more than 2 transient ischemic attack

• Blood pressure > 160/95

• Modification in medications treating for blood pressure during the last 3 months before enrollment

• Complete absence of clinical or biological symptoms

• Weight > 87 kg or < 35 kg