Phase 2
Completed N=14
Safety Study of Replagal® Therapy in Children With Fabry Disease
Source: ClinicalTrials.gov NCT01363492 ↗Enrolled (actual)
14
Serious AEs
0.0%
Results posted
May 2014
Primary outcomePrimary: Number of Serious Adverse Event (SAE) — 0 events
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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Serious Adverse Event (SAE) |
— | — |
| PRIMARY Number of Treatment Emergent Adverse Event (TEAE) |
166 | — |
| PRIMARY Development of IgG Anti-Agalsidase Alfa Antibody |
1 | — |
| PRIMARY Change From Baseline in Heart Rate Variability Parameter SDNN |
10.46 | — |
| PRIMARY Change From Baseline in Heart Rate Variability Parameter rMSSD |
1.46 | — |
| PRIMARY Change From Baseline in Heart Rate Variability Parameter pNN50 |
-4.13 | — |
| SECONDARY Change From Baseline in LVMI |
0.16 | — |
| SECONDARY Change From Baseline in MFS |
-0.62 | — |
| SECONDARY Change From Baseline in Plasma Gb3 |
-5.71 | — |
| SECONDARY Change From Baseline in Urine Gb3 |
-1403.25 | — |
Eligibility Criteria
Inclusion Criteria
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)
Exclusion Criteria
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.
Data sourced from ClinicalTrials.gov (NCT01363492). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.