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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00487630
Recruitment Status : Unknown
Verified June 2007 by Assistance Publique - Hôpitaux de Paris.
Recruitment status was:  Recruiting
First Posted : June 18, 2007
Last Update Posted : June 18, 2007
Information provided by:
Assistance Publique - Hôpitaux de Paris

Tracking Information
First Submitted Date  ICMJE June 15, 2007
First Posted Date  ICMJE June 18, 2007
Last Update Posted Date June 18, 2007
Study Start Date  ICMJE June 2005
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE
 (submitted: June 15, 2007)
Left ventricular mass [ Time Frame: 2 years ]
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: June 15, 2007)
Posterior wall thickness, interventricular thickness, ECG, creatinaemia, urinary protein / creatinine ratio, microalbuminuria, urinary Gb3 level [ Time Frame: 2 years ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease
Official Title  ICMJE 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
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.
Detailed 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
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Fabry Disease
Intervention  ICMJE Drug: recombinant alpha-galactosidase A
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: June 15, 2007)
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2009
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Female
Ages  ICMJE 15 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT00487630
Other Study ID Numbers  ICMJE AOM-01-076
PHRC National 2001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE Assistance Publique - Hôpitaux de Paris
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: 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
PRS Account Assistance Publique - Hôpitaux de Paris
Verification Date June 2007

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP