Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Last updated: January 10, 2019
Sponsor: Alexion Pharmaceuticals
Overall Status: Completed

Phase

2/3

Condition

Wolman Disease

Cholesterol Ester Storage Disease (Cesd)

Treatment

N/A

Clinical Study ID

NCT01371825
LAL-CL03
  • Ages < 24
  • All Genders

Study Summary

This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency. Eligible participants received once-weekly (qw) infusions of sebelipase alfa for up to 5 years.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Participant's parent or legal guardian provided written informed consent/permissionprior to any study procedures.

  • Male or female child with documented decreased LAL activity relative to the normalrange of the laboratory performing the assay or documented result of molecular genetictesting (2 mutations) confirming a diagnosis.

  • Growth failure with onset before 6 months of age.

Exclusion

Exclusion Criteria:

  • Clinically important concurrent disease or comorbidities.

  • Had received an investigational product other than sebelipase alfa within 14 daysprior to the first dose.

  • Participant was older than 24 months of age.

  • Myeloablative preparation, or other systemic pre-transplant conditioning, forhematopoietic stem cell or liver transplant.

  • Previous hematopoietic stem cell or liver transplant.

  • Known hypersensitivity to eggs.

Study Design

Total Participants: 9
Study Start date:
May 04, 2011
Estimated Completion Date:
January 03, 2018

Study Description

LAL Deficiency is a rare autosomal-recessive lipid storage disorder that is caused by a marked decrease or almost complete absence of LAL, leading to the accumulation of lipids, predominately cholesteryl esters and triglycerides, in various tissues and cell types. In the liver, accumulation of lipids leads to hepatomegaly, liver dysfunction, and hepatic failure. Although a single disease, LAL Deficiency presents as a clinical continuum with 2 major phenotypes, Cholesteryl Ester Storage Disease (CESD) and Wolman Disease.

Early-onset LAL Deficiency (Wolman Disease) is extremely rare, with an estimated incidence of less than 2 lives per million. It is characterized by profound malabsorption, growth failure, and hepatic failure, and is usually fatal in the first year of life.

Connect with a study center

  • Cairo, 11771
    Egypt

    Site Not Available

  • Grenoble, 38700
    France

    Site Not Available

  • Paris, 75015
    France

    Site Not Available

  • University of Mainz

    Mainz,
    Germany

    Site Not Available

  • empty

    Mumbai,
    India

    Site Not Available

  • empty

    New Delhi,
    India

    Site Not Available

  • Dublin, 1
    Ireland

    Site Not Available

  • Azienda Ospedaliera Universita di Padova

    Padova,
    Italy

    Site Not Available

  • Dept of Medical Genetics, King Faisal Specialist Hospital

    Riyadh,
    Saudi Arabia

    Site Not Available

  • National Taiwan University Hospital

    Taipei,
    Taiwan

    Site Not Available

  • empty

    Ankara,
    Turkey

    Site Not Available

  • London, SE1 7EH
    United Kingdom

    Site Not Available

  • Manchester, M13 9WL
    United Kingdom

    Site Not Available

  • Irvine, California 92697
    United States

    Site Not Available

  • North Shore Hospital Long Island Jewish Health System

    Manhasset, New York 11030
    United States

    Site Not Available

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