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NCT02292654 · ClinicalTrials.gov registry record · Phase 1
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
A Phase 1 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 1
- Development phase
- 20
- Enrollment target
NCT02292654: Completed Phase 1 study, sponsored by Genzyme, a Sanofi Company.
NCT02292654 is a Phase 1 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 20 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (67% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02292654, a Phase 1 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 20 participants
- Enrollment target
Study Summary
Primary Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks. Secondary Objective: To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.
Primary Outcome
TEAEs were defined as adverse events (AEs) that occurred or worsened during the on-treatment period (time from the start of investigational medicinal product \[IMP\]) administration until end of study (i.e. up to 64 weeks).
Interventions
- DRUG Olipudase alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2015-05-01 |
| Est. Completion | 2019-12-09 |
| Phase | Phase 1 |
What the finished NCT02292654 record still lists
NCT02292654 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 20 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (67% lower).
The record links to 0 conditions, and to 1 intervention - of which Olipudase alfa is the first listed.
NCT02292654 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02292654 about?
NCT02292654 is a clinical study titled "Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency". Primary Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks. Secondary Objective: To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase al...
What is the current status of trial NCT02292654?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 20 participants. The study started on 2015-05-01. Estimated completion is 2019-12-09.
What interventions are being tested in trial NCT02292654?
The interventions under investigation include: Olipudase alfa (DRUG).
Who is sponsoring clinical trial NCT02292654?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02292654's enrollment target sits among peer trials
20 1629th of 2000 higher than 285 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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