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A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01275066 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 177

Last updated 2014-07-07

Study results available
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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.

Conditions

  • MPS IV A

Interventions

DRUG

BMN 110 Weekly

BMN 110 Weekly: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.

DRUG

Placebo

Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week.

DRUG

BMN 110 Every Other Week

BMN 110 Every Other Week: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks.

Sponsors & Collaborators

Principal Investigators

  • Debra Lounsbury · BioMarin Pharmaceutical

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-02-28
Primary Completion
2012-08-31
Completion
2012-08-31

Countries

  • United States
  • Argentina
  • Brazil
  • Canada
  • Colombia
  • Denmark
  • France
  • Germany
  • Italy
  • Japan
  • Netherlands
  • Portugal
  • Qatar
  • Saudi Arabia
  • South Korea
  • Taiwan
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01275066 on ClinicalTrials.gov