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

Lysosomal Acid Lipase Deficiency Clinical Trial

Official title:

An Open Label Multicenter Extension Study to Evaluate the Long-term Efficacy and Safety of SBC-102 in Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01473875
• Other study ID #: LAL-CL05
• Status: Terminated
• Phase: Phase 2/Phase 3
• First received: November 10, 2011
• Last updated: February 17, 2017
• Start date: November 2011
• Est. completion date: January 2015

Study information

• Verified date: February 2017
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 10
• Est. completion date: January 2015
• Est. primary completion date: December 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

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

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

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

Exclusion Criteria:

• Clinically important concurrent disease

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

• Previous hematopoietic stem cell transplant.

Related Conditions & MeSH terms

• Lysosomal Acid Lipase Deficiency
• Wolman Disease

Intervention

Drug:
• SBC-102
SBC-102 is a recombinant human lysosomal acid lipase (rhLAL). The investigational medicinal product is an enzyme replacement therapy intended for treatment of patients with LAL Deficiency.

Locations

Country	Name	City	State
France	Hopital Necker Enfants Malades	Paris
United Kingdom	St. Mary's Hospital, Central Manchester University Hospitals	Manchester

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

Countries where clinical trial is conducted

France,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall survival		12 months
Secondary	Survival rates at periodic intervals and median survival time.		3 years
Secondary	Long-term safety of SBC-102 in children with growth failure due to LAL Deficiency		3 years

External Links

•   Alexion Website