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

NCT ID: NCT00487630

Last Updated: 2007-06-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE4

Total Enrollment

34 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-06-30

Study Completion Date

2009-06-30

Brief Summary

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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.

Detailed Description

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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

Conditions

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Fabry Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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recombinant alpha-galactosidase A

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

15 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role lead

Principal Investigators

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Dominique P GERMAIN, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

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

Locations

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

Paris, , France

Site Status RECRUITING

Countries

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France

Central Contacts

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Dominique P GERMAIN, MD, PhD

Role: CONTACT

[email protected]
+33156092306

Karelle BENISTAN, MD

Role: CONTACT

[email protected]
+33156092802

Other Identifiers

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PHRC National 2001

Identifier Type: -

Identifier Source: secondary_id

AOM-01-076

Identifier Type: -

Identifier Source: org_study_id