Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
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ClinicalTrials.gov Identifier: NCT01488097 |
Recruitment Status
:
Active, not recruiting
First Posted
: December 8, 2011
Results First Posted
: May 9, 2016
Last Update Posted
: March 6, 2017
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Cholesterol Ester Storage Disease(CESD) Lysosomal Acid Lipase Deficiency | Drug: SBC-102 (sebelipase alfa) | Phase 2 |
Cholesteryl Ester Storage Disease (CESD) is the late onset phenotype for Lysosomal Acid Lipase (LAL) Deficiency, a Lysosomal Storage Disorder, which also has an early onset phenotype known as Wolman Disease that primarily affects infants. CESD can present in childhood but often goes unrecognized until adulthood when the underlying pathology is advanced. Many of the signs and symptoms are common to patients with other liver conditions.
CESD is an autosomal recessive genetic condition and is characterized by hepatomegaly, persistently abnormal liver function tests (LFTs) and type II hyperlipidemia. Splenomegaly and evidence of mild hypersplenism may affect some patients. Untreated, CESD may lead to fibrosis, cirrhosis, liver failure and death.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 8 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open Label Multicenter Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 in Adult Subjects With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency Who Previously Received Treatment in Study LAL-CL01 |
Study Start Date : | November 2011 |
Actual Primary Completion Date : | March 2014 |
Estimated Study Completion Date : | June 2017 |

Arm | Intervention/treatment |
---|---|
Experimental: Cohort 1
Weekly IV infusions of Dose A of SBC-102 (sebelipase alfa) and bi-weekly IV infusions of Dose B or Dose C of SBC-102 (sebelipase alfa)
|
Drug: SBC-102 (sebelipase alfa)
Weekly IV infusions Dose A of SBC-102 (sebelipase alfa) and bi-weekly infusions of Dose B or Dose C of SBC-102 (sebelipase alfa)
Other Name: Kanuma
|
Experimental: Cohort 2
Weekly IV infusions of Dose B of SBC-102 (sebelipase alfa) and bi-weekly infusions of Dose B or Dose C of SBC-102 (sebelipase alfa)
|
Drug: SBC-102 (sebelipase alfa)
Weekly IV infusions Dose B of SBC-102 (sebelipase alfa) and bi-weekly infusions of Dose B or Dose C of SBC-102 (sebelipase alfa)
Other Name: Kanuma
|
Experimental: Cohort 3
Weekly IV infusions of Dose C of SBC-102 (sebelipase alfa) and bi-weekly infusions of Dose C of SBC-102 (sebelipase alfa)
|
Drug: SBC-102 (sebelipase alfa)
Weekly IV infusions Dose C of SBC-102 (sebelipase alfa) and bi-weekly infusions of Dose C of SBC-102 (sebelipase alfa)
Other Name: Kanuma
|
- ALT and AST Changes From Baseline [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]Changes from baseline (study LAL-CL01) to specific post-treatment time points in study LAL-CL04 for alanine aminotransferase (ALT) and aspartate aminotransferase (AST).
- Change From Baseline in Liver Volume [ Time Frame: From baseline (study LAL-CL04) to week 10 or 12, week 24, week 52, week 104 ]Change in liver volume (in multiples of normal [MN]) from the LAL-CL04 baseline to the indicated post-treatment time point in study LAL-CL04, as assessed by MRI
- Change From Baseline in Liver Fat Content [ Time Frame: From baseline (study LAL-CL04) to week 10 or 12, week 24, week 52, week 104 ]Percentage change in liver fat content from the LAL-CL04 baseline to the indicated post-treatment time point in study LAL-CL04, as assessed by multi-echo gradient-echo MRI
- GGT and ALP Changes From Baseline [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]Changes from baseline (study LAL-CL01) to specific post-treatment time points in study LAL-CL04 for gamma glutamyltransferase (GGT) and alkaline phosphatase (ALP).
- Lipid Changes From Baseline [ Time Frame: From baseline (study LAL-CL01) to week 10 or 12, week 24, week 52, week 104 ]Change from baseline in total cholesterol (Total-C), high density lipoprotein cholesterol (HDL-C), low density lipoprotein cholesterol (LDL-C), and triglycerides (TG).
- Serum Ferritin Change From Baseline [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]
- High Sensitivity C-reactive Protein (Hs-CRP) Change From Baseline [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]

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Ages Eligible for Study: | 18 Years to 65 Years (Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Subject received all 4 scheduled doses of SBC-102 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 liver function or lipid panel tests

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01488097
United States, California | |
Eureka, California, United States | |
San Francisco, California, United States | |
United States, Minnesota | |
Minneapolis, Minnesota, United States | |
Canada, Ontario | |
Sudbury, Ontario, Canada | |
Czech Republic | |
Prague, Czech Republic | |
France | |
Paris, France | |
United Kingdom | |
Leeds, United Kingdom | |
Salford, United Kingdom |
Additional Information:
Publications of Results:
Responsible Party: | Alexion Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT01488097 History of Changes |
Other Study ID Numbers: |
LAL-CL04 |
First Posted: | December 8, 2011 Key Record Dates |
Results First Posted: | May 9, 2016 |
Last Update Posted: | March 6, 2017 |
Last Verified: | January 2017 |
Keywords provided by Alexion Pharmaceuticals:
Enzyme Replacement Therapy (ERT) Lysosomal Storage Disease Late Onset Lysosomal Acid Lipase (LAL) Deficiency Acid cholesteryl ester hydrolase deficiency, type 2 Acid lipase disease |
Cholesterol ester hydrolase deficiency LAL Deficiency LIPA Deficiency Wolman disease |
Additional relevant MeSH terms:
Wolman Disease Cholesterol Ester Storage Disease Lipidoses Lipid Metabolism, Inborn Errors Metabolism, Inborn Errors |
Genetic Diseases, Inborn Lysosomal Storage Diseases Infant, Newborn, Diseases Lipid Metabolism Disorders Metabolic Diseases |