Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Patients With Lysosomal Acid Lipase Deficiency

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Synageva BioPharma Corp.
ClinicalTrials.gov Identifier:
NCT01307098
First received: March 1, 2011
Last updated: June 2, 2014
Last verified: June 2014

March 1, 2011
June 2, 2014
May 2011
January 2012   (final data collection date for primary outcome measure)
Safety and Tolerability of SBC-102 (sebelipase alfa) [ Time Frame: 4 weeks ] [ Designated as safety issue: Yes ]
The safety and tolerability of weekly infusions of SBC-102 (sebelipase alfa) 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.
Safety and Tolerability of SBC-102 [ Time Frame: 4 weeks ] [ Designated as safety issue: Yes ]
The safety and tolerability of weekly 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.
Complete list of historical versions of study NCT01307098 on ClinicalTrials.gov Archive Site
Pharmacokinetics of SBC-102 (sebelipase alfa) [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]
Characterize the pharmacokinetics of SBC-102 (sebelipase alfa) delivered by IV infusion after single and multiple doses.
Pharmacokinetics of SBC-102 [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]
Characterize the pharmacokinetics of SBC-102 delivered by IV infusion after single and multiple doses.
Not Provided
Not Provided
 
Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Patients With Lysosomal Acid Lipase Deficiency
An Open Label Multicenter Study to Evaluate the Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Patients With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency

This is the first clinical study of SBC-102 (sebelipase alfa) for the treatment of LAL Deficiency. It is an open label dose escalation study in adult patients with liver dysfunction due to Lysosomal Acid Lipase (LAL) Deficiency and will examine three doses of SBC-102 (sebelipase alfa). 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.

Disease Risk In Families:

  • 25 per million incidence
  • Autosomal recessive disorder, LAL deficiency is carried on chromosome 10
  • Parents with an affected son or daughter have a 1 in 4 chance of having another affected child
Interventional
Phase 1
Allocation: Non-Randomized
Endpoint Classification: Safety 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)
  • Drug: SBC-102 (sebelipase alfa)
    Weekly IV infusions Dose B of SBC-102 (sebelipase alfa)
  • Drug: SBC-102 (sebelipase alfa)
    Weekly IV infusions Dose C of SBC-102 (sebelipase alfa)
  • Experimental: Cohort 1
    Cohort 1: Weekly IV infusions of Dose A of SBC-102 (sebelipase alfa)
    Intervention: Drug: SBC-102 (sebelipase alfa)
  • Experimental: Cohort 2
    Cohort 2: Weekly IV infusions of Dose B of SBC-102 (sebelipase alfa)
    Intervention: Drug: SBC-102 (sebelipase alfa)
  • Experimental: Cohort 3
    Cohort 3: Weekly IV 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.
 
Completed
9
January 2012
January 2012   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Male or female patients ≥ 18 and ≤ 65 years of age
  • Documented decreased LAL activity
  • Evidence of liver involvement

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
  • AST and/or ALT persistently elevated > 3xULN at screening
  • Previous hemopoietic bone marrow or liver transplant
  • Current history of alcohol abuse
Both
18 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Czech Republic,   France,   United Kingdom
 
NCT01307098
LAL-CL01
Yes
Synageva BioPharma Corp.
Synageva BioPharma Corp.
Not Provided
Not Provided
Synageva BioPharma Corp.
June 2014

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