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

NCT00487630 | Unknown Phase 4 DMC

• 2 other identifiers: AOM-01-076PHRC National 2001
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Fabry Disease

Keywords

Heterozygous females; Cardiomyopathy

Outcome Measures

Primary Outcomes (1)

• Left ventricular mass

  2 years

Secondary Outcomes (1)

• Posterior wall thickness, interventricular thickness, ECG, creatinaemia, urinary protein / creatinine ratio, microalbuminuria, urinary Gb3 level

  2 years

Interventions

recombinant alpha-galactosidase A DRUG

Eligibility Criteria

• Age: 15 Years+
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Centre de reference de la maladie de Fabry et des maladies hereditaires du tissu conjonctif. Assistance Publique - Hôpitaux de Paris

Paris, France

RECRUITING

MeSH Terms

Conditions

Fabry Disease; Cardiomyopathies

Interventions

agalsidase alfa

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders; Heart Diseases

Study Officials

• Dominique P GERMAIN, MD, PhD

  Centre de reference de la maladie de Fabry et des maladies hereditaires du tissu conjonctif. Assistance Publique Hopitaux de Paris

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Dominique P GERMAIN, MD, PhD

CONTACT

+33156092306; dominique.germain@egp.aphp.fr

Karelle BENISTAN, MD

CONTACT

+33156092802; karelle.benistan@egp.aphp.fr

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: June 15, 2007
• First Posted: June 18, 2007
• Study Start: June 1, 2005
• Study Completion: June 1, 2009
• Last Updated: June 18, 2007

Record last verified: 2007-06

Locations

FR (1)