Adults 18 to 65, any sex, with Cholesterol Ester Storage Disease(CESD) or Lysosomal Acid Lipase Deficiency. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs)Primary· Screening up to Day 52
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
TEAEs
Group
Value
95% CI
Sebelipase Alfa 0.35 mg/kg
1
Sebelipase Alfa 1 mg/kg
3
Sebelipase Alfa 3 mg/kg
3
SAEs
Group
Value
95% CI
Sebelipase Alfa 0.35 mg/kg
0
Sebelipase Alfa 1 mg/kg
0
Sebelipase Alfa 3 mg/kg
0
IRRs
Group
Value
95% CI
Sebelipase Alfa 0.35 mg/kg
0
Sebelipase Alfa 1 mg/kg
0
Sebelipase Alfa 3 mg/kg
0
TEAEs Leading to Study Discontinuation
Group
Value
95% CI
Sebelipase Alfa 0.35 mg/kg
0
Sebelipase Alfa 1 mg/kg
0
Sebelipase Alfa 3 mg/kg
0
Adverse events — posted to ClinicalTrials.gov
Time frame: Screening up to Day 52.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This was the first clinical study of SBC-102 (sebelipase alfa) for the treatment of Lysosomal Acid Lipase (LAL) Deficiency. It was an open-label dose escalation study in adult participants with liver dysfunction due to LAL Deficiency and was designed to examine 3 doses of sebelipase alfa. The targeted number for this study was 9 evaluable participants.
Publications & conference data
5 peer-reviewed publications reference this trial (live from Europe PMC):
NCT06015750 — Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia
· Phase 4
· withdrawn
NCT07352423 — Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN2230 in Healthy Participants
· Phase 1
· recruiting
NCT07221838 — A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab
· Phase 4
· recruiting
NCT07413250 — Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis o
· active not recruiting
NCT07308574 — Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS
· Phase 4
· recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Alexion Pharmaceuticals, Inc.
Last refreshed: 11 December 2018
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01307098.