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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03370653 | A Study in MPS VI to Assess Safety and Efficacy of Odiparcil | PHASE2 | COMPLETED | 20 | — | — | Dec 30, 2017 | Oct 22, 2019 | Nov 1, 2019 | 4 | France, Germany +2 |
Changes in physical examination and vital signs
Change from baseline in laboratory safety tests (coagulation, liver enzymes and crystalluria) 12-lead-ECG and bone biomarkers.
Incidence of AEs/SAEs, patient withdrawals from study due to AEs/SAEs,
Change from Baseline in ECG
| Arm | Type | Description |
|---|---|---|
| Double-blind - odiparcil 1000 mg per day | EXPERIMENTAL | 2 tablets of odiparcil 250 mg per os, twice daily (BID) |
| Double-blind - odiparcil 500 mg per day | EXPERIMENTAL | 1 tablet of placebo and 1 tablet of odiparcil 250 mg per os, twice daily (BID) |
| Double-blind - placebo | PLACEBO_COMPARATOR | 2 tablets of placebo per os, twice daily (BID) |
| Open Label - odiparcil 1000 mg per day | EXPERIMENTAL | 2 tablets of odiparcil 250 mg per os, twice daily (BID) |
| Name | Type | Description |
|---|---|---|
| Odiparcil | DRUG | Investigational product: odiparcil 250 mg tablets |
| Placebo | OTHER | Comparator: placebo tablets similar to odiparcil 250 mg tablets |
1. Male or female gender. 2. Age ≥16 years. 3. Diagnosis of MPS VI, demonstrated by a reduced Arylsulfatase B (ARSB) activity relative to the normal range of the laboratory performing the assay in either white blood cells or fibroblast culture or confirmation of two known disease causing mutations i...