Phase 2 N=9 Treatment

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

Cholesterol Ester Storage Disease(CESD) · Lysosomal Acid Lipase Deficiency · LAL-Deficiency

Bottom Line

View on ClinicalTrials.gov: NCT01307098 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Dec 2018

Primary outcome: Primary: Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) — 1; 3; 3; 0 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Sebelipase alfa 0.35 mg/kg (Drug); Sebelipase alfa 1 mg/kg (Drug); Sebelipase alfa 3 mg/kg (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Alexion Pharmaceuticals, Inc.
Primary completion: Jan 2012

Outcome Measures

Outcome	Result	p-value
PRIMARY Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs)	1; 3; 3; 0; 0; 0	—

Summary

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.

Eligibility Criteria

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 circumstances
Clinically significant abnormal values on laboratory screening tests, other than liver function or lipid panel tests
Aspartate aminotransferase and/or alanine aminotransferase persistently elevated > 3x upper limit of normal at screening
Previous hemopoietic bone marrow or liver transplant
Current history of alcohol abuse

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01307098). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.