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Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01515956 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2017-08-10

Study results available
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Summary

This open-label Phase 2 study will evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age at the time of administration of the first dose of study drug, diagnosed with MPS IVA (Morquio A Syndrome) for up to 208 weeks.

Conditions

  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
  • MPS IVA

Interventions

DRUG

BMN 110

Patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/wk over a period of approximately 4 hours every week for up to 208 weeks.

Sponsors & Collaborators

Principal Investigators

  • Debra Lounsbury · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-10-31
Primary Completion
2016-02-29
Completion
2016-02-29

Countries

  • United States
  • Italy
  • Taiwan
  • 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 NCT01515956 on ClinicalTrials.gov