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

This study has been terminated.
(Study has been merged with NCT01371825)
Sponsor:
Information provided by (Responsible Party):
Synageva BioPharma Corp.
ClinicalTrials.gov Identifier:
NCT01473875
First received: November 10, 2011
Last updated: January 18, 2013
Last verified: January 2013
  Purpose

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.


Condition Intervention Phase
Lysosomal Acid Lipase Deficiency
Wolman Disease
Drug: SBC-102
Phase 2
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Synageva BioPharma Corp.:

Primary Outcome Measures:
  • Overall survival [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Survival rates at periodic intervals and median survival time. [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]
  • Long-term safety of SBC-102 in children with growth failure due to LAL Deficiency [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 10
Study Start Date: November 2011
Estimated Study Completion Date: January 2015
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: SBC-102
SBC-102 Weekly IV infusions of SBC-102
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.

Detailed 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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01473875

Locations
France
Hopital Necker Enfants Malades
Paris, France
United Kingdom
St. Mary's Hospital, Central Manchester University Hospitals
Manchester, United Kingdom
Sponsors and Collaborators
Synageva BioPharma Corp.
  More Information

No publications provided

Responsible Party: Synageva BioPharma Corp.
ClinicalTrials.gov Identifier: NCT01473875     History of Changes
Other Study ID Numbers: LAL-CL05
Study First Received: November 10, 2011
Last Updated: January 18, 2013
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
France: Conseil National de l'Ordre des Médecins

Keywords provided by Synageva BioPharma Corp.:
Wolman Disease
Acid Lipase Disease Deficiency, type 2
Cholesteryl Ester Storage Disease (CESD)
Early Onset Lysosomal Acid Lipase Deficiency (Wolman Disease)
Wolman Disease (early onset LAL Deficiency)
Related Disorders:
Non-alcoholic Fatty Liver Disease (NAFLD)
Alcoholic Liver Disease
Niemann-Pick Disease (NPD) Type C
LIPA
Wolman Phenotype
Acid Lipase Deficiency
Acid Cholesteryl Hydrolase
Cholesteryl Ester Hydrolase Deficiency
LAL Deficiency
Late Onset Lysosomal Acid Lipase Deficiency (CESD)
Non-alcoholic Steatohepatitis (NASH)
Cryptogenic Cirrhosis
Chanarin Dorfman Syndrome

Additional relevant MeSH terms:
Wolman Disease
Cholesterol Ester Storage Disease
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Lipidoses
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on November 19, 2014