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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
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
This is an extension study to the first clinical study of SBC-102 (sebelipase alfa) for the treatment of LAL Deficiency. It is an open label study in adult patients with liver dysfunction due to Lysosomal Acid Lipase (LAL) Deficiency. This study will assess the long-term safety, tolerability, and efficacy of SBC-102. The targeted number for this study is 9 evaluable subjects.

Condition or disease Intervention/treatment Phase
Cholesterol Ester Storage Disease(CESD) Lysosomal Acid Lipase Deficiency Drug: SBC-102 (sebelipase alfa) Phase 2

Detailed Description:

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 Design

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
Primary Completion Date : March 2014
Estimated Study Completion Date : June 2017


Arms and Interventions

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


Outcome Measures

Primary Outcome Measures :
  1. 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).


Secondary Outcome Measures :
  1. 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

  2. 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

  3. 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).

  4. 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).

  5. Serum Ferritin Change From Baseline [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]
  6. 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 ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations

Information from the National Library of Medicine

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


Locations
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
Sponsors and Collaborators
Alexion Pharmaceuticals
More Information

Additional Information:
Publications:
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