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NCT01275066 · ClinicalTrials.gov registry record · Phase 3
A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
A Phase 3 study, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 3
- Development phase
- 177
- Enrollment target
NCT01275066: Completed Phase 3 study, sponsored by BioMarin Pharmaceutical.
NCT01275066 is a Phase 3 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 177 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (77% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01275066, a Phase 3 study, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 177 participants
- Enrollment target
Study Summary
This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome). There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) may be a potential new treatment option for MPS IVA patients. BMN 110 is administered to MPS IVA patients by IV infusion, allowing cellular uptake by the mannose-6-phosphate receptor and transportation to the lysosomes. This enzyme uptake into the lysosomes is hypothesized to promote increased catabolism of keratan sulfate (KS) in tissue macrophages, hyaline cartilage, other connective tissues, and heart valve, and reduce the progressive accumulation of KS which is responsible for the clinical manifestations of the disorders.
Interventions
- DRUG Placebo
- DRUG BMN 110 Weekly
- DRUG BMN 110 Every Other Week
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 177 participants |
| Start Date | 2011-02 |
| Est. Completion | 2012-08 |
| Phase | Phase 3 |
What the finished NCT01275066 record still lists
NCT01275066 is an interventional study that assigns participants to a tested intervention. The registered 177 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (77% lower).
The record links to 0 conditions, and to 3 interventions - of which Placebo is the first listed.
NCT01275066 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01275066 about?
NCT01275066 is a clinical study titled "A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)". This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome). There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement the...
What is the current status of trial NCT01275066?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 177 participants. The study started on 2011-02. Estimated completion is 2012-08.
What interventions are being tested in trial NCT01275066?
The interventions under investigation include: Placebo (DRUG), BMN 110 Weekly (DRUG), BMN 110 Every Other Week (DRUG).
Who is sponsoring clinical trial NCT01275066?
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 NCT01275066's enrollment target sits among peer trials
177 1438th of 2000 higher than 563 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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