Safety Study of Replagal® Therapy in Children With Fabry Disease

Completed · Phase 2

Conditions studied: Fabry Disease

In brief

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).

Key facts

Study ID

NCT01363492

Run by

Shire

People needed

15

Starts

2011-05-12

Expected to finish

2013-04-17

Last updated by the study team

2021-06-09

Who can join

Age: 7 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients must meet all of the following criteria to be enrolled in this study.
• 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.
• The patient is 7 to <18 years of age
• The patient is ERT-naïve
• 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
• 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)

You may not qualify if…

• Patients who meet any of the following criteria will be excluded from the study.
• 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
• 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.
• Otherwise unsuitable for the study, in the opinion of the Investigator.

Where it is running

• Emory Division of Medical Genetics — Decatur, Georgia, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Baylor University Medical Center — Dallas, Texas, United States
• University of Utah Hospital — Salt Lake City, Utah, United States
• O & O Alpan LLC — Fairfax, Virginia, United States

Full record on ClinicalTrials.gov

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