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NCT02193867 · ClinicalTrials.gov registry record · Phase 2
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
A Phase 2 study, sponsored by Alexion Pharmaceuticals.
- Terminated
- Registry status
- Phase 2
- Development phase
- 10
- Enrollment target
NCT02193867: Clinical Trial Phase 2 study, sponsored by Alexion Pharmaceuticals.
NCT02193867 is a Phase 2 study that was terminated before completion, run by Alexion Pharmaceuticals. The registered enrollment target is 10 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (92% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02193867, a Phase 2 study, was terminated before completion, sponsored by Alexion Pharmaceuticals.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 10 participants
- Enrollment target
Study Summary
This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.
Primary Outcome
The number of participants experiencing severe TEAEs is presented for participants who received sebelipase alfa in this open-label study. Adverse events were obtained through spontaneous reporting or elicited by specific questioning or examination of the participant's parent or legal guardian. An adverse event was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in a participant, whether or not causally related to administration of study drug. Adver
Interventions
- DRUG Sebelipase Alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2014-06-06 |
| Est. Completion | 2018-10-30 |
| Phase | Phase 2 |
Why NCT02193867 stopped before completion
NCT02193867 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (92% lower).
The record links to 0 conditions, and to 1 intervention - of which Sebelipase Alfa is the first listed.
NCT02193867 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02193867 about?
NCT02193867 is a clinical study titled "Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency". This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.
What is the current status of trial NCT02193867?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 10 participants. The study started on 2014-06-06. Estimated completion is 2018-10-30.
What interventions are being tested in trial NCT02193867?
The interventions under investigation include: Sebelipase Alfa (DRUG).
Who is sponsoring clinical trial NCT02193867?
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 NCT02193867's enrollment target sits among peer trials
10 1968th of 2000 higher than 12 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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