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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00633139 | Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) | PHASE1 | COMPLETED | 13 | — | — | Jan 22, 2007 | Nov 25, 2008 | Jun 14, 2021 | 1 | Denmark |
Change (percent change) in GMFM is measured from baseline to end of study (Week 52). GMFM is measured using GMFM-88. The GMFM-88 item scores can be summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score is between 0 (minimal) to 3 (maximum). The total GMFM-88 score is between 0 (minimal) to 264 (maximum). Relative changes in GMFM are calculated as percentage change from baseline divided by the age difference in months between first and last visit. The GMFM score decreases over time, which, indicates that the disease worsened over time. Score over time (SOT), data mentioned over mean represents the adjusted mean.
Changes in Mullen's Scales of Early Learning are measured from baseline to end of study (Week 52) using Mullen's Scales of Early Learning. T scores, percentile ranks, and age equivalents can be computed for the four scales separately (visual reception, fine motor, expressive language, and receptive language). Relative change is calculated as percentage change from baseline divided by the age-difference in months between first and last visit. When Mullen's score decreases over time, it indicates the disease worsened over time. Data mentioned over mean represents the adjusted mean.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | Cohort 1: 50 U/kg Recombinant human Arylsulfatase A (rhASA) |
| Cohort 2 | EXPERIMENTAL | Cohort 2: 100 U/kg Recombinant human Arylsulfatase A (rhASA) |
| Cohort 3 | EXPERIMENTAL | Cohort 3: 200 U/kg Recombinant human Arylsulfatase A (rhASA) |
| Name | Type | Description |
|---|---|---|
| Recombinant human Arylsulfatase A (rhASA) | BIOLOGICAL | intravenous infusion, every other week for 26 weeks |
Inclusion Criteria: The patients from the Phase I trial must meet the following criteria to be enrolled in the study. * Subject's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (trial-related activities are any procedures that ...