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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. 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

Tracking Information
First Submitted Date  ICMJE December 22, 2011
First Posted Date  ICMJE January 24, 2012
Results First Submitted Date  ICMJE April 13, 2017
Results First Posted Date  ICMJE July 6, 2017
Last Update Posted Date August 10, 2017
Study Start Date  ICMJE October 2011
Actual Primary Completion Date February 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 7, 2017)
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
Original Primary Outcome Measures  ICMJE
 (submitted: January 23, 2012)
Descriptive summary of clinical safety assessments [ Time Frame: Up to 52 weeks ]
Safety will be determined by the following factors: Number and severity of adverse events in participants. Clinically significant changes in any of the following assessments from Baseline: Vital Signs, echocardiogram, ECG, immunogenicity tests, physical and neurological examinations, standard clinical laboratory tests, concomitant medications, and cervical spine radiography.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 7, 2017)
  • 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.
  • 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).
Original Secondary Outcome Measures  ICMJE
 (submitted: January 23, 2012)
  • Change in Urinary Keratan Sulfate measures over time [ Time Frame: Baseline, and weeks: 2, 4, 8, 13, 26, 39, and 52/ETV ]
    To Evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce the urinary KS levels in MPS IVA patients less than 5 years.
  • Change in patient growth over time [ Time Frame: Baseline and Weeks: 13, 26, 39, and 52/ETV ]
    Changes in growth over time will be assessed using anthropometric measurements and radiographs of lower extremities.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Official Title  ICMJE 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)
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
Intervention  ICMJE 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
Study Arms  ICMJE Experimental: BMN 110 Weekly
Intervention: Drug: BMN 110
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.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 23, 2012)
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE February 2016
Actual Primary Completion Date February 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 5 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Italy,   Taiwan,   United Kingdom,   United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT01515956
Other Study ID Numbers  ICMJE MOR-007
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party BioMarin Pharmaceutical
Study Sponsor  ICMJE BioMarin Pharmaceutical
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Debra Lounsbury BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date July 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP