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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00319046 | Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease | PHASE3 | COMPLETED | 42 | — | — | Feb 1, 2006 | Jul 1, 2010 | Feb 4, 2025 | - | — |
Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging. Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the 'worst' within-patient value not more than 2 days after the end of treatment was used to impute the missing value.
Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging. Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the 'worst' within-patient value not more than 2 days after the end of treatment was used to impute the missing value.
| Arm | Type | Description |
|---|---|---|
| Open-label miglustat | EXPERIMENTAL | Oral administration of miglustat 100 mg t.i.d. for a period of 2 years |
| Name | Type | Description |
|---|---|---|
| Miglustat | DRUG | Oral capsules containing miglustat 100 mg, administered three times daily (t.i.d.) |
Inclusion Criteria: 1. Males or females aged 18 years or older 2. Type 1 Gaucher disease, diagnosed by glucocerebrosidase assay or molecular analysis of the glucocerebrosidase gene. 3. Treatment with ERT for at least 3 years, with a stable dose regimen for at least the last 6 months. 4. Clinically ...