Phase 2
N=17
Replagal Enzyme Replacement Therapy for Children With Fabry Disease
Fabry Disease
Bottom Line
View on ClinicalTrials.gov: NCT00084084 ↗Enrolled (actual)
17
Serious AEs
14.3%
Results posted
Oct 2013
Primary outcome: Primary: Patients Who Experienced At Least One Adverse Event (AE) — 17 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Agalsidase alfa (Drug)
- Age
- Pediatric · 7+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- Jun 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Patients Who Experienced At Least One Adverse Event (AE) |
17 | — |
| SECONDARY Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 81 |
245282 | — |
| SECONDARY Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 133 |
295779 | — |
| SECONDARY Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 159 |
218078 | — |
| SECONDARY Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 315/341 |
213193 | — |
| SECONDARY Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 81 |
3173 | — |
| SECONDARY Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 133 |
3842 | — |
| SECONDARY Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 159 |
3842 | — |
| SECONDARY Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 315/341 |
3568 | — |
Summary
Primary Objective(s):
* To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease
* To assess the effect of Replagal on heart rate variability in patients 7 to 17 years of age
Secondary Objective(s):
* To determine the pharmacokinetics of Replagal at baseline and after the initiation of enzyme replacement therapy (ERT)
* To determine exploratory measurements of efficacy including renal function (ie, estimated glomerular filtration rate [eGFR] and creatinine clearance), clinical outcomes (in Cohorts 1 and 2), and sweating and left ventricular mass index (LVMI) (Cohort 1, Phase 1 only)
Eligibility Criteria
Inclusion Criteria
1a. For Cohort 1 (both phases):
- Patients must have completed all study requirements and assessments for Study TKT023 less than 30 (+/-7) days prior to enrolling in Study TKT029 and must have no safety or medical issues that contraindicate participation.
OR
1b. For Cohort 2:
- The patient is between 7 and 17 years of age at the time of informed consent, inclusive.
- The patient must be ERT-naive.
- The patient is a hemizygous male with Fabry disease as confirmed by a deficiency of alpha-galactosidase A activity measured in serum, leukocytes, or fibroblasts. Male patients who do not already have a documented deficiency of alpha-galactosidase A activity will provide a blood sample during screening for determination of alpha-galactosidase A activity level in their serum.
OR
- The patient is a heterozygous female or hemizygous male with Fabry disease as confirmed by a mutation of the alpha-galactosidase A gene. Patients who do not already have a documented mutation of the alpha-galactosidase A gene will provide a blood sample during screening for genotyping.
- 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 guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee (IRB/IEC) approved informed concent form after all relevant aspects of the study have been explained and discussed with the child and the child's parent(s) or legal guardian(s).
Exclusion Criteria
Patients who meet any of the following criteria are not eligible for this study:
- Patient and/or the patient's parent(s) or legal guardian(s) are unable to understand the nature, scope, and possible consequences of the study.
- Patient is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator or the medical monitor.
Data sourced from ClinicalTrials.gov (NCT00084084). 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.