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Completed

NCT Number: NCT01515956

Study of BMN 110 in Pediatric Patients < 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.

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Key information

Conditions

Mucopolysaccharidosis IVA Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases MPS IVA Metabolic Diseases Metabolism, Inborn Errors Morquio A Syndrome Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis IV Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

Up to 5 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Monza, Italy

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

BMN 110

Drug

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: N-acetylgalactosamine-6-sulfatase, N-acetylgalactosamine-6-sulfate, sulfatase, galactose-6-sulfatase, GALNS, enzyme replacement therapy, ERT

Primary outcomes

  1. 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

    Time frame: 52 weeks

    Number of Participants Experiencing Adverse Events

Secondary outcomes

  1. Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures

    Time frame: Baseline to Week 52

    Percent Change from Baseline to Week 52 for Urinary Keratan Sulfate measures.

  2. Change From Baseline in Normalized Growth Rate Z-Scores

    Time frame: 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).

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study title

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)

Important dates

Study start
2011
Primary completion
2016
Study completion
2016
First posted
Jan 24, 2012
Registry last updated
Aug 10, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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