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

NCT01307098 | Completed Phase 1 Results DMC

• 1 other identifier: LAL-CL01
• Study Type: interventional
• Target: 9
• Locations: 4 countries
• Sites: 7

Brief 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.

Conditions

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

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs)

  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 regardless of causality is located in the Reported Adverse Events module.

  Screening up to Day 52

Study Arms (3)

Sebelipase alfa 0.35 mg/kg

EXPERIMENTAL

Cohort 1: Participants were administered once weekly (qw) infusions of 0.35 mg/kg sebelipase alfa.

Drug: Sebelipase alfa 0.35 mg/kg

Sebelipase alfa 1 mg/kg

EXPERIMENTAL

Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa.

Drug: Sebelipase alfa 1 mg/kg

Sebelipase alfa 3 mg/kg

EXPERIMENTAL

Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa.

Drug: Sebelipase alfa 3 mg/kg

Interventions

Sebelipase alfa 0.35 mg/kg DRUG

Sebelipase alfa is a recombinant human lysosomal acid lipase.

Also known as: Kanuma, SBC-102

Sebelipase alfa 0.35 mg/kg

Sebelipase alfa 1 mg/kg DRUG

Sebelipase alfa is a recombinant human lysosomal acid lipase.

Also known as: Kanuma, SBC-102

Sebelipase alfa 1 mg/kg

Sebelipase alfa 3 mg/kg DRUG

Sebelipase alfa is a recombinant human lysosomal acid lipase.

Also known as: Kanuma, SBC-102

Sebelipase alfa 3 mg/kg

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female participants ≥ 18 and ≤ 65 years of age
• Documented decreased LAL activity
• Evidence of liver involvement

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (7)

Unknown Facility

Stanford, California, 94305, United States

Location

Unknown Facility

New York, New York, 10029, United States

Location

Unknown Facility

Pittsburgh, Pennsylvania, 15213, United States

Location

Unknown Facility

Prague, 12000, Czechia

Location

Unknown Facility

Paris, 75743, France

Location

Unknown Facility

Cambridge, CB20QQ, United Kingdom

Location

Unknown Facility

Manchester, M139WL, United Kingdom

Location

Related Publications (1)

• Balwani M, Breen C, Enns GM, Deegan PB, Honzik T, Jones S, Kane JP, Malinova V, Sharma R, Stock EO, Valayannopoulos V, Wraith JE, Burg J, Eckert S, Schneider E, Quinn AG. Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease. Hepatology. 2013 Sep;58(3):950-7. doi: 10.1002/hep.26289. Epub 2013 Mar 28.

  PMID: 23348766 RESULT

Related Links

• Website

MeSH Terms

Conditions

Cholesterol Ester Storage Disease; Wolman Disease; Lysosomal Storage Diseases

Interventions

Sebelipase alfa

Condition Hierarchy (Ancestors)

Lipidoses; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Infant, Newborn, Diseases

Results Point of Contact

• Title: Alexion Pharmaceuticals Inc.
• Organization: Alexion Pharmaceuticals Inc.

clinicaltrials@alexion.com

855-752-2356

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 1, 2011
• First Posted: March 2, 2011
• Study Start: April 25, 2011
• Primary Completion: January 6, 2012
• Study Completion: January 6, 2012
• Last Updated: December 11, 2018
• Results First Posted: December 11, 2018

Record last verified: 2018-12

Locations

FR (1); CZ (1); US (3); GB (2)