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

A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

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.

Study Overview

• Status: Completed
• Conditions: Morquio A Syndrome; MPS IVA; Mucopolysaccharidosis IVA
• Intervention / Treatment: Drug: BMN 110

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 2

Contacts and Locations

Study Locations

• Italy
  • Monza, Italy
• Taiwan
  • Taipei, Taiwan
• United Kingdom
  • Central Manchester, United Kingdom
• United States
  • California
    • Oakland, California, United States
  • New York
    • Manhasset, New York, United States

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 5 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BMN 110 Weekly	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.; Other Names: ERT; N-acetylgalactosamine-6-sulfatase; galactose-6-sulfatase; GALNS; enzyme replacement therapy; N-acetylgalactosamine-6-sulfate; sulfatase

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Number of Participants Experiencing Adverse Events	52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures	Percent Change from Baseline to Week 52 for Urinary Keratan Sulfate measures.	Baseline to Week 52
Change From Baseline in Normalized Growth Rate Z-Scores	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).	Baseline to Week 52

Collaborators and Investigators

• Sponsor: BioMarin Pharmaceutical

Publications and helpful links

General Publications

• Jones SA, Bialer M, Parini R, Martin K, Wang H, Yang K, Shaywitz AJ, Harmatz P. Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y. Pediatr Res. 2015 Dec;78(6):717-22. doi: 10.1038/pr.2015.169. Epub 2015 Sep 2.

Study record dates

Study Major Dates

• Study Start: October 1, 2011
• Primary Completion (Actual): February 1, 2016
• Study Completion (Actual): February 1, 2016

Study Registration Dates

• First Submitted: December 22, 2011
• First Submitted That Met QC Criteria: January 23, 2012
• First Posted (Estimate): January 24, 2012

Study Record Updates

• Last Update Posted (Actual): August 10, 2017
• Last Update Submitted That Met QC Criteria: July 12, 2017
• Last Verified: July 1, 2017

More Information

Terms related to this study

• Keywords: enzyme replacement therapy; Lysosomal Storage Disorder; GALNS; Mucopolysaccharidosis IVA; MPS IVA; Morquio A Syndrome; LSD; N-acetylgalactosamine-6-sulfatase; galactose-6-sulfatase; ERT; MPS IVA Type A; N-acetylgalactosamine-6-sulfate; sulfatase; Mucopolysaccharidosis IVA type A
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Disease; Genetic Diseases, Inborn; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Syndrome; Mucopolysaccharidoses; Mucopolysaccharidosis IV
• Other Study ID Numbers: MOR-007