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NCT01371825 · ClinicalTrials.gov registry record · Phase 2
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
A Phase 2 study, sponsored by Alexion Pharmaceuticals.
- Completed
- Registry status
- Phase 2
- Development phase
- 9
- Enrollment target
NCT01371825: Completed Phase 2 study, sponsored by Alexion Pharmaceuticals.
NCT01371825 is a Phase 2 study that has completed, run by Alexion Pharmaceuticals. The registered enrollment target is 9 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (93% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01371825, a Phase 2 study, has completed, sponsored by Alexion Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 9 participants
- Enrollment target
Study Summary
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency. Eligible participants received once-weekly (qw) infusions of sebelipase alfa for up to 5 years.
Primary Outcome
The primary efficacy endpoint was the percentage of participants (%) in the PES who survived to at least 12 months of age.
Interventions
- DRUG Sebelipase alfa (SBC-102)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2011-05-04 |
| Est. Completion | 2018-01-03 |
| Phase | Phase 2 |
What the finished NCT01371825 record still lists
NCT01371825 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 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (93% lower).
The record links to 0 conditions, and to 1 intervention - of which Sebelipase alfa (SBC-102) is the first listed.
NCT01371825 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01371825 about?
NCT01371825 is a clinical study titled "Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency". This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency. Eligible participants received once-weekly (qw) infusions of sebelipase alfa for up to 5 years.
What is the current status of trial NCT01371825?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 9 participants. The study started on 2011-05-04. Estimated completion is 2018-01-03.
What interventions are being tested in trial NCT01371825?
The interventions under investigation include: Sebelipase alfa (SBC-102) (DRUG).
Who is sponsoring clinical trial NCT01371825?
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 NCT01371825's enrollment target sits among peer trials
9 1989th of 2000 higher than 11 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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