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

Study Identifier:
LAL-CL01
ClinicalTrials.gov Identifier:
NCT01307098
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: Sebelipase alfa 0.35 mg/kg
  • Drug: Sebelipase alfa 1 mg/kg
  • Drug: Sebelipase alfa 3 mg/kg
Date
Apr 2011 - Jan 2012
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 65 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Inclusion Criteria:
  • Male or female participants ≥ 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
  • Aspartate aminotransferase and/or alanine aminotransferase persistently elevated \> 3x upper limit of normal at screening
  • Previous hemopoietic bone marrow or liver transplant
  • Current history of alcohol abuse
Sex
Female & Male
Age
18 - 65 Years

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: Sebelipase alfa 0.35 mg/kg
  • Drug: Sebelipase alfa 1 mg/kg
  • Drug: Sebelipase alfa 3 mg/kg
Date
Apr 2011 - Jan 2012
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 65 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Inclusion Criteria:
  • Male or female participants ≥ 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
  • Aspartate aminotransferase and/or alanine aminotransferase persistently elevated \> 3x upper limit of normal at screening
  • Previous hemopoietic bone marrow or liver transplant
  • Current history of alcohol abuse

Protocol Summary

This was the first clinical study of SBC-102 (sebelipase alfa) for the treatment of Lysosomal Acid Lipase (LAL) Deficiency. It was an open-label dose escalation study in adult participants with liver dysfunction due to LAL Deficiency and was designed to examine 3 doses of sebelipase alfa. The targeted number for this study was 9 evaluable participants.

Trial Locations

Location
Status
Location
Stanford, California, United States, 94305
Status
N/A
Location
New York, New York, United States, 10029
Status
N/A
Location
Pittsburgh, Pennsylvania, United States, 15213
Status
N/A
Location
Prague, Czech Republic, 12000
Status
N/A
Location
Paris, France, 75743
Status
N/A
Location
Cambridge, United Kingdom, CB20QQ
Status
N/A
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