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NCT01307098 · ClinicalTrials.gov registry record · Phase 1
Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency
A Phase 1 study, sponsored by Alexion Pharmaceuticals.
- Completed
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
NCT01307098: Completed Phase 1 study, sponsored by Alexion Pharmaceuticals.
NCT01307098 is a Phase 1 study that has completed, run by Alexion Pharmaceuticals. The registered enrollment target is 9 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01307098, a Phase 1 study, has completed, sponsored by Alexion Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 9 participants
- Enrollment target
Study 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.
Primary Outcome
Safety and tolerability of sebelipase alfa was primarily assessed by monitoring the number of participants reporting treatment-emergent adverse events (TEAEs), including serious adverse events, and infusion-related reactions (IRRs). The number of participants who discontinued from the study due to a TEAE is also presented. An IRR was defined as any adverse event that occurred between the start of the infusion and 4 hours after completion of the infusion and was assessed by the Investigator as at
Interventions
- DRUG Sebelipase alfa 0.35 mg/kg
- DRUG Sebelipase alfa 1 mg/kg
- DRUG Sebelipase alfa 3 mg/kg
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2011-04-25 |
| Est. Completion | 2012-01-06 |
| Phase | Phase 1 |
What the finished NCT01307098 record still lists
NCT01307098 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower).
The record links to 0 conditions, and to 3 interventions - of which Sebelipase alfa 0.35 mg/kg is the first listed.
NCT01307098 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01307098 about?
NCT01307098 is a clinical study titled "Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency". 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 target...
What is the current status of trial NCT01307098?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2011-04-25. Estimated completion is 2012-01-06.
What interventions are being tested in trial NCT01307098?
The interventions under investigation include: Sebelipase alfa 0.35 mg/kg (DRUG), Sebelipase alfa 1 mg/kg (DRUG), Sebelipase alfa 3 mg/kg (DRUG).
Who is sponsoring clinical trial NCT01307098?
This trial is sponsored by Alexion Pharmaceuticals, which has 162 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01307098's enrollment target sits among peer trials
9 1955th of 2000 higher than 42 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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