Safety Study of Replagal® Therapy in Children With Fabry Disease

Fabry Disease Clinical Trial

Official title:

An Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement Therapy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01363492
• Other study ID #: HGT-REP-084
• Status: Completed
• Phase: Phase 2
• Start date: May 12, 2011
• Est. completion date: April 17, 2013

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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).

Description:

In 2008, a change in the agalsidase alfa drug substance manufacturing process was made. There are no changes to the drug product formulation, manufacturing site, manufacturing process, or container closure.

An agalsidase alfa bioreactor manufacturing process (agalAF1) utilizing animal component-free media replaced the previous roller bottle (RB) process.

This study will evaluate the safety of Replagal AF, manufactured using the new bioreactor 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: April 17, 2013
• Est. primary completion date: April 17, 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 7 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Biological:
• Replagal (agalsidase alfa)
0.2 mg/kg administered over 40 minutes every other week (EOW)

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Serious Adverse Event (SAE)		Baseline to week 55
Primary	Number of Treatment Emergent Adverse Event (TEAE)		Baseline to week 55
Primary	Development of IgG Anti-Agalsidase Alfa Antibody	Reflects development of Anti-Agalsidase antibodies post baseline	Baseline to Week 55
Primary	Change From Baseline in Heart Rate Variability Parameter SDNN		Baseline to week 55
Primary	Change From Baseline in Heart Rate Variability Parameter rMSSD		Baseline to week 55
Primary	Change From Baseline in Heart Rate Variability Parameter pNN50		Baseline to week 55
Secondary	Change From Baseline in LVMI		Baseline to week 55
Secondary	Change From Baseline in MFS		Baseline to week 55
Secondary	Change From Baseline in Plasma Gb3		Baseline to week 55
Secondary	Change From Baseline in Urine Gb3		Baseline to week 55