Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 is a Phase 3 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 177 participants.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01275066
The ClinicalTrials.gov registry entry for NCT01275066 describes a study currently listed as completed, categorized as Phase 3. The registered enrollment target is 177 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is BioMarin Pharmaceutical, which has 80 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 3 interventions - of which Placebo is the first listed.
NCT01275066 reports 0 study locations.
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
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.