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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00074971 | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease | PHASE3 | COMPLETED | 58 | — | — | Oct 1, 1999 | Dec 1, 2004 | Dec 4, 2013 | 20 | United States, France +3 |
| NCT00196716 | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease | PHASE2 | COMPLETED | 21 | — | — | Jun 1, 2003 | Mar 1, 2007 | Apr 3, 2015 | 4 | Czechia, Estonia +2 |
| NCT00074958 | A Study of Fabrazyme in Pediatric Patients With Fabry Disease | PHASE2 | COMPLETED | 16 | — | — | Oct 1, 2002 | Jul 1, 2005 | Apr 2, 2015 | 7 | United States, France +2 |
Kidney biopsies were taken at Baseline, Week 24, and Week 96 and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was evaluated by pathologists for the total number of vessels with GL-3 accumulation on an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe).
Skin biopsies were taken at Baseline, Week 24 and Week 48 and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was evaluated by pathologists for the total number of vessels with GL-3 accumulation on an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe).
| Arm | Type | Description |
|---|---|---|
| Fabrazyme | EXPERIMENTAL | Open-label study. Patients received 1.0 mg/kg Fabrazyme every two weeks for approximately six months followed by 0.3 mg/kg Fabrazyme every two weeks for approximately 18 months. |
| Name | Type | Description |
|---|---|---|
| Fabrazyme (agalsidase beta) | DRUG | - |
Inclusion Criteria: * Patients must have successfully completed the previous double-blind study (AGAL-1-002-98) * Patients must provide written informed consent prior to study participation * Female patients must have a negative pregnancy test prior to each dosing and use a medically accepted metho...