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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01722526 | Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients | PHASE1 | COMPLETED | 5 | — | — | Mar 1, 2013 | Jan 1, 2014 | Jul 30, 2015 | 2 | United States, United Kingdom |
| Arm | Type | Description |
|---|---|---|
| Recombinant human acid sphingomyelinase | EXPERIMENTAL | Participants will receive rhASM of an initial dose of 0.1 mg/kg, followed by several dose escalations, as tolerated, up to 3.0 mg/kg. All doses are given 2 weeks apart. |
| Name | Type | Description |
|---|---|---|
| Recombinant human acid sphingomyelinase | DRUG | Administered intravenously every 2 weeks for 26 weeks |
Inclusion Criteria: * Patients with documented non-neuronopathic acid sphingomyelinase deficiency * The patient has a diffusing capacity of carbon monoxide (DLco) \>20% and ≤80% of the predicted normal value. * The patient has a spleen volume ≥6 multiples of normal(MN). A partial splenectomy will b...