At a glance
ClinicalIndex Comparison Record- ✓Cohort 1: Completed Study TKT023 within 30 days prior to enrollment with no safety/medical contraindications
- ✓Cohort 2: Age 7-17 years at informed consent
- ✓Cohort 2: ERT-naive (no prior enzyme replacement therapy)
- ✓Fabry disease confirmed by alpha-galactosidase A enzyme deficiency (males) or GLA gene mutation (males or females)
- ✕Unable to understand the nature, scope, and consequences of the study
- ✕Unable to comply with protocol schedule or procedures, or unlikely to complete the study
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
An Open Label Clinical Trial of Replagal Enzyme Replacement Therapy In Children With Fabry Disease Who Have Completed Study TKT023 or Who Are Naive to Enzyme Replacement Therapy
In Brief
A Phase 2 clinical trial evaluating Agalsidase alfa for Fabry Disease. Completed, enrolled 17 participants across 14 sites in 2 countries.
Detailed 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)
Study Details
Timeline
Interventions
0.2 mg/kg agalsidase alfa administered by IV infusion over 40 (+/- 10) minutes every other week for 52 weeks, with periodic reassessments for study continuation beyond 52 weeks