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NCT01757184 · ClinicalTrials.gov registry record · Phase 3
Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
A Phase 3 study of Lysosomal Acid Lipase Deficiency, sponsored by Alexion Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 66
- Enrollment target
- 20
- Study locations
NCT01757184: Completed Phase 3 study of Lysosomal Acid Lipase Deficiency, sponsored by Alexion Pharmaceuticals.
NCT01757184 is a Phase 3 study of Lysosomal Acid Lipase Deficiency that has completed, run by Alexion Pharmaceuticals. The registered enrollment target is 66 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (91% lower). The trial reports 20 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01757184, a Phase 3 study of Lysosomal Acid Lipase Deficiency, has completed, sponsored by Alexion Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 66 participants
- Enrollment target
- 20
- Study locations
Study Summary
This Phase 3 study evaluated the efficacy and safety of 1 milligram/kilogram (mg/kg) intravenous (IV) infusions of SBC-102 (sebelipase alfa) administered every other week (qow) in participants with late onset lysosomal acid lipase deficiency (LAL-D) (cholesteryl ester storage disease \[CESD\]). Late-onset LAL-D is an underappreciated cause of cirrhosis, liver failure and dyslipidemia. There is currently no standard treatment for LAL-D other than supportive care. Enzyme replacement therapy may be a potential new treatment option for LAL-D participants.
Primary Outcome
Alanine aminotransferase (ALT) normalization was defined as an abnormal baseline value (ALT \> the age- and gender-specific upper limit of normal \[ULN\] provided by the central laboratory performing the assay) that becomes normal (\< ULN). Alanine aminotransferase normalization was evaluated at the end of the Double-blind Period (the last double-blind assessment) and at the end of the Open-label Period (last open-label assessment). Baseline for the Open-label Period was defined relative to the
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Sebelipase Alfa
Study Locations (20)
Other
- - Córdoba
- - Brisbane
- - New Lambton
- - Parkville
- - Perth
- - Zagreb
- - Olomouc
- - Prague
- - Paris
New York
- - Buffalo
- - Manhasset
- - New York
California
- - Palo Alto
- - San Francisco
Arizona
- - Tucson
Delaware
- - Wilmington
Illinois
- - Chicago
Massachusetts
- - Boston
Ohio
- - Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 66 participants |
| Start Date | 2013-01-22 |
| Est. Completion | 2018-12-11 |
| Phase | Phase 3 |
What the finished NCT01757184 record still lists
NCT01757184 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 66 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (91% lower).
The record links to 1 condition, with Lysosomal Acid Lipase Deficiency appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT01757184 names 20 study sites across 9 states, led by Other, New York, California.
Frequently Asked Questions
What is clinical trial NCT01757184 about?
NCT01757184 is a clinical study titled "Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency". This Phase 3 study evaluated the efficacy and safety of 1 milligram/kilogram (mg/kg) intravenous (IV) infusions of SBC-102 (sebelipase alfa) administered every other week (qow) in participants with late onset lysosomal acid lipase deficiency (LAL-D) (cholesteryl ester storage disease \[CESD\]). Lat...
What is the current status of trial NCT01757184?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 66 participants. The study started on 2013-01-22. Estimated completion is 2018-12-11.
What conditions does trial NCT01757184 study?
This clinical trial studies the following conditions: Lysosomal Acid Lipase Deficiency.
What interventions are being tested in trial NCT01757184?
The interventions under investigation include: Placebo (DRUG), Sebelipase Alfa (DRUG).
Who is sponsoring clinical trial NCT01757184?
This trial is sponsored by Alexion Pharmaceuticals, which has 162 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01757184 being conducted?
This trial has 20 study locations across Arizona, California, Delaware, Illinois, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT01757184's enrollment target sits among peer trials
66 1797th of 2000 higher than 204 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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