Safety Study of Replagal® Therapy in Children With Fabry Disease
An Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement Therapy
1 other identifier
interventional
15
1 country
5
Brief 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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started May 2011
5 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 31, 2011
CompletedStudy Start
First participant enrolled
May 12, 2011
CompletedFirst Posted
Study publicly available on registry
June 1, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 17, 2013
CompletedStudy Completion
Last participant's last visit for all outcomes
April 17, 2013
CompletedResults Posted
Study results publicly available
May 20, 2014
CompletedJune 9, 2021
May 1, 2021
1.9 years
March 31, 2011
March 25, 2014
May 24, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (6)
Number of Serious Adverse Event (SAE)
Baseline to week 55
Number of Treatment Emergent Adverse Event (TEAE)
Baseline to week 55
Development of IgG Anti-Agalsidase Alfa Antibody
Reflects development of Anti-Agalsidase antibodies post baseline
Baseline to Week 55
Change From Baseline in Heart Rate Variability Parameter SDNN
Baseline to week 55
Change From Baseline in Heart Rate Variability Parameter rMSSD
Baseline to week 55
Change From Baseline in Heart Rate Variability Parameter pNN50
Baseline to week 55
Secondary Outcomes (4)
Change From Baseline in LVMI
Baseline to week 55
Change From Baseline in MFS
Baseline to week 55
Change From Baseline in Plasma Gb3
Baseline to week 55
Change From Baseline in Urine Gb3
Baseline to week 55
Study Arms (1)
Replagal 0.2 mg/kg every other week (EOW)
EXPERIMENTALInterventions
0.2 mg/kg administered over 40 minutes every other week (EOW)
Eligibility Criteria
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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Shirelead
Study Sites (5)
Emory Division of Medical Genetics
Decatur, Georgia, 30033, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Baylor University Medical Center
Dallas, Texas, 75246, United States
University of Utah Hospital
Salt Lake City, Utah, 84132, United States
O & O Alpan LLC
Fairfax, Virginia, 22152, United States
Related Publications (1)
Goker-Alpan O, Longo N, McDonald M, Shankar SP, Schiffmann R, Chang P, Shen Y, Pano A. An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naive to enzyme replacement therapy. Drug Des Devel Ther. 2016 May 25;10:1771-81. doi: 10.2147/DDDT.S102761. eCollection 2016.
PMID: 27307708DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Shire
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 31, 2011
First Posted
June 1, 2011
Study Start
May 12, 2011
Primary Completion
April 17, 2013
Study Completion
April 17, 2013
Last Updated
June 9, 2021
Results First Posted
May 20, 2014
Record last verified: 2021-05