Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00701415 | A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms | PHASE3 | COMPLETED | 31 | — | — | Sep 1, 2008 | Jun 1, 2015 | Jun 29, 2016 | 12 | United States, Argentina +7 |
Skin biopsies were taken at Baseline, Week 52, Week 156 and Week 260 or early withdrawal and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was scored for GL-3 accumulation on a severity score-scale of none, mild, moderate, severe (0-1-2-3). Scores are categorized as normal (score = 0) or abnormal (score = 1, 2 or 3). Data was summarized in terms of number of participants with none/trace, mild, moderate and severe biopsy scores.
| Arm | Type | Description |
|---|---|---|
| Fabrazyme 0.5 mg/kg | EXPERIMENTAL | Fabrazyme 0.5 mg/kg was administered every 2 weeks (up to 131 infusion) up to 260 weeks, the total infusion time was not less than 45 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks. |
| Fabrazyme 1.0 mg/kg | EXPERIMENTAL | Fabrazyme 1.0 mg/kg was administered every 4 weeks (up to 66 infusion) up to 260 weeks, the total infusion time was not less than 90 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks. |
| Name | Type | Description |
|---|---|---|
| Agalsidase beta | BIOLOGICAL | Powder for concentrate for solution for infusion 1.0 mg/kg/4 weeks |
Inclusion Criteria: * The participant and/or participant's parent(s)/legal guardian(s) must provide written informed assent/consent prior to any protocol-related procedures being performed. * The participant must had a confirmed diagnosis of Fabry disease as documented by leukocyte α-Galactosidase ...