Status:

COMPLETED

A Study of Fabrazyme in Pediatric Patients With Fabry Disease

Lead Sponsor:

Genzyme, a Sanofi Company

Conditions:

Fabry Disease

Eligibility:

All Genders

7-15 years

Phase:

PHASE2

Brief Summary

People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. This enzyme helps to break down and remove certain types of fatt...

Eligibility Criteria

Inclusion

  • Inclusion criteria:
  • Patient or legal guardian must provide written informed consent
  • Patients must have a clinical diagnosis of Fabry disease and active Fabry disease (clinical signs and symptoms)
  • Patients must be at least 7 years of age but no older than 15 years of age at time of enrollment
  • Patients must be Tanner Stage ≤ III
  • Female patients must have a negative pregnancy test prior to each infusion and use a medically accepted form of contraception throughout the study
  • Exclusion Criteria:
  • Patient has a clinically significant organic disease (with the exception of symptoms relating to Fabry disease) that in the opinion of the investigator would preclude participation in the trial
  • Patient has participated in a study employing investigational drug within 30 days of the start of this study
  • Patient has received prior treatment with enzyme replacement therapy
  • Patient is unable to comply with the clinical protocol

Exclusion

    Key Trial Info

    Start Date :

    October 1 2002

    Trial Type :

    INTERVENTIONAL

    Allocation :

    ACTUAL

    End Date :

    July 1 2005

    Estimated Enrollment :

    16 Patients enrolled

    Trial Details

    Trial ID

    NCT00074958

    Start Date

    October 1 2002

    End Date

    July 1 2005

    Last Update

    April 2 2015

    Active Locations (7)

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    Page 1 of 2 (7 locations)

    1

    University of Arizona

    Tucson, Arizona, United States, 85724

    2

    Hopital Edouard Herriot

    Lyon, France, Cedex 03

    3

    Hopital de la Timone Enfants

    Marseille, France, Cedex 05

    4

    Hopital Europeen Georges Pompidou

    Paris, France, Cedex 15