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Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

NCT01697319 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2016-01-12

Study results available
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Summary

The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks.

Conditions

  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
  • MPS IVA

Interventions

DRUG

BMN 110

Drug will be delivered through a 4 hour (approximate) IV infusion at a dosage amount of 2.0 mg/kg/week for up to 144 weeks of treatment.

Sponsors & Collaborators

Principal Investigators

  • Celeste Decker, M.D. · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-08-31
Primary Completion
2014-10-31
Completion
2014-10-31

Countries

  • United States
  • Germany
  • United Kingdom

Study Locations

More Related Trials

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 NCT01697319 on ClinicalTrials.gov