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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01515956
Recruitment Status : Completed
First Posted : January 24, 2012
Results First Posted : July 6, 2017
Last Update Posted : August 10, 2017
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis IVA Morquio A Syndrome MPS IVA Drug: BMN 110 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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)
Study Start Date : October 2011
Actual Primary Completion Date : February 2016
Actual Study Completion Date : February 2016

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

Primary Outcome Measures :
  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 Outcome Measures :
  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).

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Ages Eligible for Study:   up to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01515956

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United States, California
Oakland, California, United States
United States, New York
Manhasset, New York, United States
Monza, Italy
Taipei, Taiwan
United Kingdom
Central Manchester, United Kingdom
Sponsors and Collaborators
BioMarin Pharmaceutical
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Study Director: Debra Lounsbury BioMarin Pharmaceutical
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01515956    
Other Study ID Numbers: MOR-007
First Posted: January 24, 2012    Key Record Dates
Results First Posted: July 6, 2017
Last Update Posted: August 10, 2017
Last Verified: July 2017
Keywords provided by BioMarin Pharmaceutical:
Morquio A Syndrome
Mucopolysaccharidosis IVA type A
Mucopolysaccharidosis IVA
Lysosomal Storage Disorder
enzyme replacement therapy
Additional relevant MeSH terms:
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Mucopolysaccharidosis IV
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases