Completed

Has Results

Replagal Enzyme Replacement Therapy for Children with Fabry Disease

WHAT IS THE PURPOSE OF THIS STUDY?

Primary Objective(s):- To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease- To assess the effect of Replagal on heart rate variability in patients 7 to 17 years of ageSecondary Objective(s):- To determine the pharmacokinetics of Replagal at baseline and after the initiation of enzyme replacement therapy (ERT)- To determine exploratory measurements of efficacy including renal function (ie, estimated glomerular filtration rate [eGFR] and creatinine clearance), clinical outcomes (in Cohorts 1 and 2), and sweating and left ventricular mass index (LVMI) (Cohort 1, Phase 1 only)

TRIAL AT A GLANCE

Condition

Fabry Disease

Study Intervention

Agalsidase alfa

Possibility of Active Treatment

This study is complete and no longer enrolling patients.

Study Type

Interventional

Phase

Phase 2

IS THIS STUDY RIGHT FOR ME?

AGE

7 to 17

Years

GENDERS

ACCEPTS HEALTHY VOLUNTEERS

No

PARTICIPATION DURATION/ REQUIREMENTS

This study is complete and no longer enrolling patients.

ENTRY CRITERIA

For Cohort 1 (both phases): Patients must have completed all study requirements and assessments for Study TKT023 less than 30 (+/-7) days prior to enrolling in Study TKT029 and must have no safety or medical issues that contraindicate participation. For Cohort 2: The patient must be ERT-naive.

For Cohort 2: The patient is a hemizygous male with Fabry disease as confirmed by a deficiency of alpha-galactosidase A activity measured in serum, leukocytes, or fibroblasts. Male patients who do not already have a documented deficiency of alpha-galactosidase A activity will provide a blood sample during screening for determination of alpha-galactosidase A activity level in their serum. OR The patient is a heterozygous female or hemizygous male with Fabry disease as confirmed by a mutation of the alpha-galactosidase A gene. Patients who do not already have a documented mutation of the alpha-galactosidase A gene will provide a blood sample during screening for genotyping.

Study Results

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results

Clinical Study Results
ClinicalTrials.gov Identifier
NCT00084084

Study Locations

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RESULTS

  • 1.

    Christus St. Patrick Hospital

    Lake Charles, LA
    United States of America

    miles

  • 2.

    Children's Physician Group

    Palm Beach Gardens, Florida
    United States of America

    miles

  • 3.

    Sacred Heart Hospital

    Allentown, Pennsylvania
    United States of America

    miles

  • 4.

    Clinical Center, National Institutes of Health

    Bethesda, Maryland
    United States of America

    miles

  • 5.

    Memorial Hospital

    Easton, MD
    United States of America

    miles

  • 6.

    East Tennessee Children's Hospital

    Knoxville, Tennessee
    United States of America

    miles

  • 7.

    Institute of Metabolic Diseases

    Dallas, TX
    United States of America

    miles

  • 8.

    The Hospital for Sick Children

    Toronto, Ontario
    Canada

    miles

  • 9.

    NYU School of Medicine

    New York, NY
    United States of America

    miles

  • 10.

    Office of Michael Cohen

    Stafford, Virginia
    United States of America

    miles

  • 11.

    University of Arizona Health Sciences Center

    Tucson, Arizona
    United States of America

    miles

  • 12.

    University of Tennessee, Health Science Center

    Memphis, TN
    United States of America

    miles

  • 13.

    Tucson Access Center of Arizona Kidney Disease Hypertension Center

    Tucson, AZ
    United States of America

    miles

  • 14.

    St. Louis Children's Hospital

    St. Louis, Missouri
    United States of America

    miles