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NCT01924845 · ClinicalTrials.gov registry record · Phase 3
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
A Phase 3 study, sponsored by BioMarin Pharmaceutical.
- Terminated
- Registry status
- Phase 3
- Development phase
- 24
- Enrollment target
NCT01924845: Clinical Trial Phase 3 study, sponsored by BioMarin Pharmaceutical.
NCT01924845 is a Phase 3 study that was terminated before completion, run by BioMarin Pharmaceutical. The registered enrollment target is 24 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01924845, a Phase 3 study, was terminated before completion, sponsored by BioMarin Pharmaceutical.
- TERMINATED
- Registry status
- Phase 3
- Development phase
- 24 participants
- Enrollment target
Study Summary
Study 701-301 is a single-arm, open-label, switchover study in patients with late-onset Pompe disease who have been receiving treatment with recombinant human acid alpha-glucosidase (rhGAA) for 48 weeks or longer. Ambulatory patients who have mild to moderate respiratory impairment will switch directly to receive BMN 701 20 mg/kg by IV infusion every other week. All participants will receive active drug. No dose of existing therapy will be missed - experimental drug is started immediately.
Primary Outcome
Pulmonary function test: Percent Predicted Maximum Inspiratory Pressure
Interventions
- DRUG BMN 701
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2014-04 |
| Est. Completion | 2016-09-12 |
| Phase | Phase 3 |
Why NCT01924845 stopped before completion
NCT01924845 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 24 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower).
The record links to 0 conditions, and to 1 intervention - of which BMN 701 is the first listed.
NCT01924845 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01924845 about?
NCT01924845 is a clinical study titled "BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)". Study 701-301 is a single-arm, open-label, switchover study in patients with late-onset Pompe disease who have been receiving treatment with recombinant human acid alpha-glucosidase (rhGAA) for 48 weeks or longer. Ambulatory patients who have mild to moderate respiratory impairment will switch direc...
What is the current status of trial NCT01924845?
This trial is currently terminated. It is a Phase 3 study. The enrollment target is 24 participants. The study started on 2014-04. Estimated completion is 2016-09-12.
What interventions are being tested in trial NCT01924845?
The interventions under investigation include: BMN 701 (DRUG).
Who is sponsoring clinical trial NCT01924845?
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 NCT01924845's enrollment target sits among peer trials
24 1943rd of 2000 higher than 49 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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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