Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102

Last updated: February 17, 2017
Sponsor: Alexion Pharmaceuticals
Overall Status: Terminated

Phase

2/3

Condition

Cholesterol Ester Storage Disease (Cesd)

Wolman Disease

Treatment

N/A

Clinical Study ID

NCT01473875
LAL-CL05
  • All Genders

Study Summary

This phase 2/3, open-label extension study will evaluate the long-term efficacy and safety of intravenous (IV) infusions of SBC-102 in children with Lysosomal Acid Lipase (LAL) Deficiency who previously received treatment with SBC-102.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Subject's parent or legal guardian provides written consent/permission prior to anystudy procedures

  • Subject completed treatment in study LAL-CL03 or Subject received treatment withSBC-102 for at least 4 months under an expanded access treatment regimen

  • Subject had no life-threatening or unmanageable study drug toxicity during treatmentwith SBC-102 under LAL-CL03 or expanded access treatment regimen.

Exclusion

Exclusion Criteria:

  • Clinically important concurrent disease

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

  • Previous hematopoietic stem cell transplant.

Study Design

Total Participants: 10
Study Start date:
November 01, 2011
Estimated Completion Date:
January 31, 2015

Study Description

Early onset LAL Deficiency is a very rare form of LAL Deficiency, with an estimated prevalence of less than 2 lives per million (Meikle et al., 1999). This form of the disease, named after the physician who first described it (Abramov et al., 1956), is the most aggressive presentation of LAL Deficiency and is characterized by gastrointestinal and hepatic manifestations including marked growth failure, malabsorption, steatorrhea, and hepatomegaly. Early onset LAL Deficiency is rapidly progressive and fatal usually within the first year of life (Assmann & Seedorf, 2001).

The primary objective of the study is to evaluate the effect of SBC-102 therapy on overall survival at 12 months of age in children with growth failure due to LAL Deficiency.

All subjects will receive repeat IV infusions of SBC-102, beginning at least 1 week after the preceding infusion in study LAL-CL03 or under an expanded access treatment regimen.

Connect with a study center

  • Hopital Necker Enfants Malades

    Paris,
    France

    Site Not Available

  • St. Mary's Hospital, Central Manchester University Hospitals

    Manchester,
    United Kingdom

    Site Not Available

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