Phase 2
N=8
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
Cholesterol Ester Storage Disease (CESD) · Lysosomal Acid Lipase Deficiency · LAL-Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT01488097 ↗Enrolled (actual)
8
Serious AEs
12.5%
Results posted
May 2016
Primary outcome: Primary: Number Of Participants Reporting TEAEs And IARs — 8; 1; 2; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- sebelipase alfa (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Jun 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number Of Participants Reporting TEAEs And IARs |
8; 1; 2; 0 | — |
| SECONDARY Changes From Baseline In ALT And AST |
-35.9; -38.1; -40.6; -42.5; -29.5; -26.7 | — |
| SECONDARY Changes From Baseline In Liver Volume |
-0.096; -0.099; -0.096; -0.176; -0.082; -0.182 | — |
| SECONDARY Changes From Baseline In Liver Fat Content |
-2.816; -2.772; -3.633; -4.348; -3.953; -4.007 | — |
| SECONDARY Changes From Baseline In GGT And ALP |
-13.6; -13.8; -14.0; -22.4; 0.1; 2.3 | — |
| SECONDARY Changes From Baseline In Serum Lipids |
-34.5; -59.3; -73.9; -60.8; -36.4; 1.1 | — |
| SECONDARY Changes From Baseline In Serum Ferritin |
-37.0; -40.1; -18.1; -22.8; 0.0; 18.0 | — |
| SECONDARY Changes From Baseline In Hs-CRP |
-1.41; -1.03; -1.40; -1.50; -1.09; -1.34 | — |
Summary
This was an extension study to Study LAL-CL01 (NCT01307098). The primary objective of the study was to evaluate the long-term safety and tolerability of sebelipase alfa in participants with liver dysfunction due to lysosomal acid lipase (LAL) deficiency.
Eligibility Criteria
Inclusion Criteria
- Participant received all 4 scheduled doses of sebelipase alfa in Study LAL-CL01 with no life-threatening or unmanageable study drug toxicity.
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 LFTs or lipid panel tests
Data sourced from ClinicalTrials.gov (NCT01488097). 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.