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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01307098 | Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency | PHASE1 | COMPLETED | 9 | — | — | Apr 25, 2011 | Jan 6, 2012 | Dec 11, 2018 | 7 | United States, Czechia +2 |
Safety and tolerability of sebelipase alfa was primarily assessed by monitoring the number of participants reporting treatment-emergent adverse events (TEAEs), including serious adverse events, and infusion-related reactions (IRRs). The number of participants who discontinued from the study due to a TEAE is also presented. An IRR was defined as any adverse event that occurred between the start of the infusion and 4 hours after completion of the infusion and was assessed by the Investigator as at least possibly related to study drug. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
| Arm | Type | Description |
|---|---|---|
| Sebelipase alfa 0.35 mg/kg | EXPERIMENTAL | Cohort 1: Participants were administered once weekly (qw) infusions of 0.35 mg/kg sebelipase alfa. |
| Sebelipase alfa 1 mg/kg | EXPERIMENTAL | Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa. |
| Sebelipase alfa 3 mg/kg | EXPERIMENTAL | Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa. |
| Name | Type | Description |
|---|---|---|
| Sebelipase alfa 0.35 mg/kg | DRUG | Sebelipase alfa is a recombinant human lysosomal acid lipase. |
| Sebelipase alfa 1 mg/kg | DRUG | Sebelipase alfa is a recombinant human lysosomal acid lipase. |
| Sebelipase alfa 3 mg/kg | DRUG | Sebelipase alfa is a recombinant human lysosomal acid lipase. |
Inclusion Criteria: * Male or female participants ≥ 18 and ≤ 65 years of age * Documented decreased LAL activity * Evidence of liver involvement Exclusion Criteria: * Clinically significant concurrent disease, serious inter-current illness, concomitant medications or other extenuating circumstanc...