Safety Study of Replagal® Therapy in Children With Fabry Disease
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01363492|
Recruitment Status : Completed
First Posted : June 1, 2011
Results First Posted : May 20, 2014
Last Update Posted : June 9, 2021
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|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease||Biological: Replagal (agalsidase alfa)||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement Therapy|
|Actual Study Start Date :||May 12, 2011|
|Actual Primary Completion Date :||April 17, 2013|
|Actual Study Completion Date :||April 17, 2013|
|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
- Number of Serious Adverse Event (SAE) [ Time Frame: Baseline to week 55 ]
- Number of Treatment Emergent Adverse Event (TEAE) [ Time Frame: Baseline to week 55 ]
- Development of IgG Anti-Agalsidase Alfa Antibody [ Time Frame: Baseline to Week 55 ]Reflects development of Anti-Agalsidase antibodies post baseline
- Change From Baseline in Heart Rate Variability Parameter SDNN [ Time Frame: Baseline to week 55 ]
- Change From Baseline in Heart Rate Variability Parameter rMSSD [ Time Frame: Baseline to week 55 ]
- Change From Baseline in Heart Rate Variability Parameter pNN50 [ Time Frame: Baseline to week 55 ]
- Change From Baseline in LVMI [ Time Frame: Baseline to week 55 ]
- Change From Baseline in MFS [ Time Frame: Baseline to week 55 ]
- Change From Baseline in Plasma Gb3 [ Time Frame: Baseline to week 55 ]
- Change From Baseline in Urine Gb3 [ Time Frame: Baseline to week 55 ]
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|Ages Eligible for Study:||7 Years to 17 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
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)
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01363492
|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|
|Study Director:||Study Director||Takeda|
|Other Study ID Numbers:||
|First Posted:||June 1, 2011 Key Record Dates|
|Results First Posted:||May 20, 2014|
|Last Update Posted:||June 9, 2021|
|Last Verified:||May 2021|
Enzyme Replacement Therapy
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lipid Metabolism Disorders