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Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

NCT01609062 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2016-02-01

Study results available
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Summary

The primary objective of this study was to evaluate the safety of a 2.0 mg/kg/week and a 4.0 mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 196 weeks. Secondary objectives were to investigate the effect of the two doses on exercise capacity for up to 196 weeks. In addition, the pharmacokinetic (PK) parameters of both doses of BMN 110 was assessed.

Conditions

  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
  • MPS IVA

Interventions

DRUG

BMN 110

Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for up to 192 weeks.

DRUG

BMN 110

Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion for 27 weeks, and will eventually transition to 2.0 mg/kg/week for up to an additional 166 weeks.

Sponsors & Collaborators

Principal Investigators

  • Adam Shaywitz, MD PhD · BioMarin Pharmaceutical

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
7 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-04-30
Primary Completion
2014-11-30
Completion
2014-11-30

Countries

  • United States
  • Canada
  • Germany
  • 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 NCT01609062 on ClinicalTrials.gov