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NCT01230801 · ClinicalTrials.gov registry record · Phase 1
Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
A Phase 1 study, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 1
- Development phase
- 22
- Enrollment target
NCT01230801: Completed Phase 1 study, sponsored by BioMarin Pharmaceutical.
NCT01230801 is a Phase 1 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 22 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (63% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01230801, a Phase 1 study, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 22 participants
- Enrollment target
Study Summary
A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.
Primary Outcome
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Interventions
- BIOLOGICAL BMN 701
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 22 participants |
| Start Date | 2011-01-17 |
| Est. Completion | 2013-03-06 |
| Phase | Phase 1 |
What the finished NCT01230801 record still lists
NCT01230801 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 22 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (63% lower).
The record links to 0 conditions, and to 1 intervention - of which BMN 701 is the first listed.
NCT01230801 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01230801 about?
NCT01230801 is a clinical study titled "Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease". A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.
What is the current status of trial NCT01230801?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 22 participants. The study started on 2011-01-17. Estimated completion is 2013-03-06.
What interventions are being tested in trial NCT01230801?
The interventions under investigation include: BMN 701 (BIOLOGICAL).
Who is sponsoring clinical trial NCT01230801?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01230801's enrollment target sits among peer trials
22 1601st of 2000 higher than 386 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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