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Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Synageva BioPharma Corp.
ClinicalTrials.gov Identifier:
NCT01488097
First received: November 26, 2011
Last updated: July 7, 2014
Last verified: July 2014

November 26, 2011
July 7, 2014
November 2011
December 2014   (final data collection date for primary outcome measure)
Long term safety of SBC-102 including incidence of adverse events [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]
The safety and tolerability of infusions of SBC-102 will be assessed by routine monitoring of patients for adverse events (AEs) and monitoring changes from baseline in physical examination findings, vital signs, clinical laboratory evaluations, immunogenicity tests and concomitant therapies.
Same as current
Complete list of historical versions of study NCT01488097 on ClinicalTrials.gov Archive Site
  • Changes in liver and spleen volume and fat content. [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • Characterize the pharmacokinetics (e.g. AUC, Cmax, T1/2) of multiple doses of SBC-102 delivered by IV infusion. [ Time Frame: Pre-dose, 10, 15, 20, 40, 60 and 90 minutes during the infusion, the end of the infusion, and at 5, 10, 20, 30, 40, 60 and 120 minutes after completion of the infusion ] [ Designated as safety issue: No ]
  • Asses pharmacodynamics of SBC-102 on specified biomarkers, including change in transaminases, serum lipids and acute phase reactants. [ Time Frame: 3 years ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
An Open Label Multicenter Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 in Adult Subjects With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency Who Previously Received Treatment in Study LAL-CL01

This is an extension study to the first clinical study of SBC-102 (sebelipase alfa) for the treatment of LAL Deficiency. It is an open label study in adult patients with liver dysfunction due to Lysosomal Acid Lipase (LAL) Deficiency. This study will assess the long-term safety, tolerability, and efficacy of SBC-102. The targeted number for this study is 9 evaluable subjects.

Cholesteryl Ester Storage Disease (CESD) is the late onset phenotype for Lysosomal Acid Lipase (LAL) Deficiency, a Lysosomal Storage Disorder, which also has an early onset phenotype known as Wolman Disease that primarily affects infants. CESD can present in childhood but often goes unrecognized until adulthood when the underlying pathology is advanced. Many of the signs and symptoms are common to patients with other liver conditions.

CESD is an autosomal recessive genetic condition and is characterized by hepatomegaly, persistently abnormal liver function tests (LFTs) and type II hyperlipidemia. Splenomegaly and evidence of mild hypersplenism may affect some patients. Untreated, CESD may lead to fibrosis, cirrhosis, liver failure and death.

Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Cholesterol Ester Storage Disease(CESD)
  • Lysosomal Acid Lipase Deficiency
  • Drug: SBC-102 (sebelipase alfa)
    Weekly IV infusions Dose A of SBC-102 (sebelipase alfa) and bi-weekly infusions of Dose B or Dose C of SBC-102 (sebelipase alfa)
  • Drug: SBC-102 (sebelipase alfa)
    Weekly IV infusions Dose B of SBC-102 (sebelipase alfa) and bi-weekly infusions of Dose B or Dose C of SBC-102 (sebelipase alfa)
  • Drug: SBC-102 (sebelipase alfa)
    Weekly IV infusions Dose C of SBC-102 (sebelipase alfa) and bi-weekly infusions of Dose C of SBC-102 (sebelipase alfa)
  • Experimental: Cohort 1
    Weekly IV infusions of Dose A of SBC-102 (sebelipase alfa) and bi-weekly IV infusions of Dose B or Dose C of SBC-102 (sebelipase alfa)
    Intervention: Drug: SBC-102 (sebelipase alfa)
  • Experimental: Cohort 2
    Weekly IV infusions of Dose B of SBC-102 (sebelipase alfa) and bi-weekly infusions of Dose B or Dose C of SBC-102 (sebelipase alfa)
    Intervention: Drug: SBC-102 (sebelipase alfa)
  • Experimental: Cohort 3
    Weekly IV infusions of Dose C of SBC-102 (sebelipase alfa) and bi-weekly infusions of Dose C of SBC-102 (sebelipase alfa)
    Intervention: Drug: SBC-102 (sebelipase alfa)
Balwani M, Breen C, Enns GM, Deegan PB, Honzík 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 Jan 24. doi: 10.1002/hep.26289. [Epub ahead of print]

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
8
January 2015
December 2014   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Subject received all 4 scheduled doses of SBC-102 in study LAL-CL01 with no life-threatening or unmanageable study drug toxicity.

Exclusion Criteria:

  • 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
Both
18 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Canada,   Czech Republic,   France,   United Kingdom
 
NCT01488097
LAL-CL04
Yes
Synageva BioPharma Corp.
Synageva BioPharma Corp.
Not Provided
Not Provided
Synageva BioPharma Corp.
July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP