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Safety Study of Replagal® Therapy in Children With Fabry Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:
NCT01363492
First received: March 31, 2011
Last updated: May 22, 2013
Last verified: May 2013
History of Changes
  Purpose

The purpose of this study is to assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT).


Condition Intervention Phase
Fabry Disease
 Biological: Replagal (agalsidase alfa)
 Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement Therapy

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Shire Human Genetic Therapies, Inc.:

Primary Outcome Measures:
  • Assess the safety of Replagal in children with Fabry disease who are naive to enzyme replacement therapy (ERT) [ Time Frame: Safety Assessments will be performed from baseline to week 55. ] [ Designated as safety issue: Yes ]
    Safety evaluations include adverse events, vital signs, physical examination, echocardiograms, neurological examination, concomitant medication usage, electrocardiogram (ECG) , clinical laboratory assessments and immunogenicity.


Secondary Outcome Measures:
  • To determine the pharmacokinetics of Replagal at baseline and after the initiation of ERT [ Time Frame: Blood samples for Pharmacokinetic (PK) analysis will be collected immediately prior to the start of the first infusion, once at Week 25, and once at Week 55. ] [ Designated as safety issue: No ]
    The individual and mean serum concentration-time profiles of agalsidase alfa will be presented with summary statistics. PK parameters will be computed where appropriate for PK assessments at Weeks 25 and 55 and compared to Week 1 PK parameters.


Enrollment: 15
Study Start Date: May 2011
Study Completion Date: April 2013
Primary Completion Date: April 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Replagal 0.2 mg/kg every other week (EOW) Biological: Replagal (agalsidase alfa)
0.2 mg/kg administered over 40 minutes every other week (EOW)
Other Name: agalsidase alfa

Detailed Description:

This study will evaluate the safety of Replagal manufactured using a new process at a dose of 0.2 mg/kg infused IV over 40 minutes, every other week (EOW) in children with Fabry disease who are 7 years to less than 18 years of age and who are naive to ERT.

  Eligibility

Ages Eligible for Study:   7 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Patients must meet all of the following criteria to be enrolled in this study.

  1. All patients must be diagnosed with Fabry disease by the following criteria:

    • Male Patients: The patient is a hemizygous male with Fabry disease as confirmed by a deficiency of alfa-galactosidase A activity measured in serum, leukocytes, or fibroblasts or has a confirmed mutation of the alfa-galactosidase-A gene.
    • Female Patients: The patient is a heterozygous female with Fabry disease as confirmed by a mutation of the alfa-galactosidase A gene.

    Note: If the diagnosis of Fabry disease is previously documented in the patient's medical record, screening tests do not need to be repeated.

  2. The patient is 7 to <18 years of age
  3. The patient is ERT-naïve
  4. Adequate general health (as determined by the Investigators) to undergo the specified phlebotomy regimen and protocol-related procedures and no safety or medical contraindications for participation
  5. The minor child must assent to participate in the protocol and the parent(s) or legally authorized representative(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee (IRB/IEC) approved informed consent form after all relevant aspects of the study have been explained and discussed with the child and the child's parent(s) or legally authorized representative(s)

Exclusion Criteria:

Patients who meet any of the following criteria will be excluded from the study.

  1. Patient and/or the patient's parent(s) or legally authorized representative(s) are unable to understand the nature, scope, and possible consequences of the study
  2. Patient is unable to comply with the protocol, eg, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator or the medical monitor.
  3. Otherwise unsuitable for the study, in the opinion of the Investigator.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01363492

Locations
United States, Georgia
Emory Division of Medical Genetics
Decatur, Georgia, United States, 30033
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Texas
Baylor University Medical Center
Dallas, Texas, United States, 75246
United States, Utah
University of Utah Hospital
Salt Lake City, Utah, United States, 84132
United States, Virginia
O & O Alpan LLC
Fairfax, Virginia, United States, 22152
Sponsors and Collaborators
Shire Human Genetic Therapies, Inc.
Investigators
Principal Investigator: Ozlem Goker-Alpan, MD O & O Alpan LLC
Principal Investigator: Nicola Longo, MD, PhD University of Utah Hospital
Principal Investigator: Raphael Schiffmann, MD Baylor Health Care System
Principal Investigator: Suma P. Shankar, MD, PhD Emory Division of Medical Genetics
Principal Investigator: Marie T. McDonald, MD Duke University
  More Information

No publications provided

Responsible Party: Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier: NCT01363492     History of Changes
Other Study ID Numbers: HGT-REP-084
Study First Received: March 31, 2011
Last Updated: May 22, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Shire Human Genetic Therapies, Inc.:
agalsidase alfa
Replagal
Enzyme Replacement Therapy

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on June 18, 2013