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Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01415427 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 173

Last updated 2021-07-21

Study results available
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Summary

This Phase 3 extension study will evaluate the long-term efficacy and safety of BMN 110 2.0 mg/kg/week and/or BMN 110 2.0 mg/kg/every other week in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).

Conditions

  • Mucopolysaccharidosis IV A
  • Morquio A Syndrome
  • MPS IVA

Interventions

DRUG

BMN 110 - Weekly

In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/qw administered over a period of approximately 4 hours once a week. In Part 2, patients will continue to receive 2.0 mg/kg of BMN 110 every week, with no placebo.

DRUG

BMN 110 - Every Other Week

In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week. Patients randomized to the 2.0 mg/kg/qow arm will receive infusions of placebo on alternating weeks, to mask active drug weeks. In Part 2, patients will receive 2.0 mg/kg of BMN 110 every week, with no placebo.

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
2011-07-31
Primary Completion
2016-06-16
Completion
2016-06-16

Countries

  • United States
  • Argentina
  • Brazil
  • Canada
  • Colombia
  • Denmark
  • France
  • Germany
  • Italy
  • Japan
  • Netherlands
  • Norway
  • Portugal
  • Qatar
  • Saudi Arabia
  • South Korea
  • Spain
  • Taiwan
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

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Entities

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 NCT01415427 on ClinicalTrials.gov