Status:

COMPLETED

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

Lead Sponsor:

Alexion Pharmaceuticals, Inc.

Conditions:

Cholesterol Ester Storage Disease (CESD)

Lysosomal Acid Lipase Deficiency

Eligibility:

All Genders

18+ years

Phase:

PHASE2

Brief Summary

This was an extension study to Study LAL-CL01 (NCT01307098). The primary objective of the study was to evaluate the long-term safety and tolerability of sebelipase alfa in participants with liver dysf...

Detailed Description

Participants who successfully received all 4 doses of sebelipase alfa in Study LAL-CL01 and opted to continue treatment in the extension study underwent screening assessments to determine study eligib...

Eligibility Criteria

Inclusion

  • Participant received all 4 scheduled doses of sebelipase alfa in Study LAL-CL01 with no life-threatening or unmanageable study drug toxicity.

Exclusion

  • Clinically significant concurrent disease, serious inter-current illness, concomitant medications or other extenuating circumstances
  • Clinically significant abnormal values on laboratory screening tests, other than LFTs or lipid panel tests

Key Trial Info

Start Date :

December 12 2011

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

June 21 2017

Estimated Enrollment :

8 Patients enrolled

Trial Details

Trial ID

NCT01488097

Start Date

December 12 2011

End Date

June 21 2017

Last Update

July 20 2018

Active Locations (10)

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Page 1 of 3 (10 locations)

1

Eureka, California, United States, 95501-320

2

Sacramento, California, United States, 95817

3

San Francisco, California, United States, 94143

4

Minneapolis, Minnesota, United States, 55455