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

Phase 2

Completed

• Conditions: Morquio A Syndrome; MPS IVA; Mucopolysaccharidosis IVA
• Interventions: Drug: BMN 110

• Registration Number: NCT01515956
• Lead Sponsor: BioMarin Pharmaceutical

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-10
• Study First Submitted: 2011-12-22
• Study First Submitted QC: 2012-01-23
• Study First Posted: 2012-01-24
• Primary Completion: 2016-02
• Study Completion: 2016-02
• Results First Submitted: 2017-04-13
• Results First Submitted QC: 2017-06-07
• Status Verified: 2017-07
• Results First Posted: 2017-07-06
• Last Update Submitted: 2017-07-12
• Last Update Posted: 2017-08-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

• Less than 5 years of age at the time of the first study drug infusion
• Documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA
• Written informed consent provided by parent or legally authorized representative after the nature of the study has been explained and prior to any research-related procedures.

Exclusion Criteria

• Previous hematopoietic stem cell transplant (HSCT).
• Previous treatment with BMN 110.
• Known hypersensitivity to any of the components of BMN 110.
• Major surgery within 3 months prior to stuy entry or planned major surgery during the 52-week treatment period.
• Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
• Concurrent disease or condition, including but not limited to symptomatic cervical spine instability, clinically significant spinal cord compression, or severe cardiac disease that would interfere with study participation or safety as determined by the Investigator.
• Any condition that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
BMN 110 Weekly	BMN 110	-

Primary Outcome Measures

Name	Time	Method
To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug Infusion	52 weeks	Number of Participants Experiencing Adverse Events

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Normalized Growth Rate Z-Scores	Baseline to Week 52	Changes in growth over time will be assessed using anthropometric measurements and radiographs of lower extremities. Z-scores are the normalized scores derived from the reference population mean and standard deviation (A positive change from baseline indicates that the population has moved closer to the reference population and represents a positive outcome).
Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures	Baseline to Week 52	Percent Change from Baseline to Week 52 for Urinary Keratan Sulfate measures.