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

NCT01307098 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2018-12-11

Study results available
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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.

Conditions

  • Cholesterol Ester Storage Disease(CESD)
  • Lysosomal Acid Lipase Deficiency
  • LAL-Deficiency

Interventions

DRUG

Sebelipase alfa 0.35 mg/kg

Sebelipase alfa is a recombinant human lysosomal acid lipase.

DRUG

Sebelipase alfa 1 mg/kg

Sebelipase alfa is a recombinant human lysosomal acid lipase.

DRUG

Sebelipase alfa 3 mg/kg

Sebelipase alfa is a recombinant human lysosomal acid lipase.

Sponsors & Collaborators

  • Alexion Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-04-25
Primary Completion
2012-01-06
Completion
2012-01-06

Countries

  • United States
  • Czechia
  • France
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01307098 on ClinicalTrials.gov