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

Fabry Disease Clinical Trial

— HEART  

Official title:

A Multicenter, Phase 4, Randomized, Controlled Study to Evaluate the Efficacy and Safety of Recombinant Alpha-Galactosidase A (Agalsidase Beta, FABRAZYME) in Heterozygous Females for Fabry Disease

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00487630
• Other study ID #: AOM-01-076
• Secondary ID: PHRC National 20
• Status: Recruiting
• Phase: Phase 4
• First received: June 15, 2007
• Last updated: June 15, 2007
• Start date: June 2005
• Est. completion date: June 2009

Study information

• Verified date: June 2007
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: Dominique P GERMAIN, MD, PhD
• Phone: +33156092306
• Email: dominique.germain[@]egp.aphp.fr
• Is FDA regulated: No
• Health authority: France: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

Description:

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 :

• left ventricular posterior wall thickness (echocardiography)

• interventricular septum thickness (echocardiography)

• tissue doppler imaging (myocardial function)

• EKG

• creatinaemia

• serum cystatin C level

• urinary protein/creatinine ratio

• microalbuminuria

• Gb3 urinary levels

Evaluation of tolerance and safety with :

• Home therapy infusions follow up

• Vitals

• Physical examination

• Adverse events

• Antibodies levels

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 34
• Est. completion date: June 2009
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 15 Years and older

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• recombinant alpha-galactosidase A

Locations

Country	Name	City	State
France	Centre de reference de la maladie de Fabry et des maladies hereditaires du tissu conjonctif. Assistance Publique - Hôpitaux de Paris	Paris

Sponsors (1)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Left ventricular mass		2 years
Secondary	Posterior wall thickness, interventricular thickness, ECG, creatinaemia, urinary protein / creatinine ratio, microalbuminuria, urinary Gb3 level		2 years