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COMPLETED Phase 3

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

NCT01757184 · View on ClinicalTrials.gov ↗

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.

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

FieldValue
Enrollment Target 66 participants
Start Date 2013-01-22
Est. Completion 2018-12-11
Phase Phase 3

Sponsor

Alexion Pharmaceuticals

109 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT01757184

The ClinicalTrials.gov registry entry for NCT01757184 describes a study currently listed as completed. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 66 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Alexion Pharmaceuticals, which has 109 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT01757184 reports 20 study locations spanning 9 distinct geographic areas — top geographies include Other, New York, California. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT01757184?

The interventions under investigation include: Placebo (DRUG), Sebelipase Alfa (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT01757184?

This trial is sponsored by Alexion Pharmaceuticals, which has 109 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial