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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01515956
First Posted: January 24, 2012
Last Update Posted: August 10, 2017
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.
Information provided by (Responsible Party):
BioMarin Pharmaceutical
December 22, 2011
January 24, 2012
April 13, 2017
July 6, 2017
August 10, 2017
October 2011
February 2016   (Final data collection date for primary outcome measure)
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
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.

Complete list of historical versions of study NCT01515956 on ClinicalTrials.gov Archive Site
  • 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).
  • 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.
Not Provided
Not Provided
 
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.
Not Provided
Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
  • MPS IVA
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
  • GALNS
  • enzyme replacement therapy
  • ERT
Experimental: BMN 110 Weekly
Intervention: Drug: BMN 110
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 ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
15
February 2016
February 2016   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
up to 5 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Italy,   Taiwan,   United Kingdom,   United States
 
 
NCT01515956
MOR-007
Yes
Not Provided
Not Provided
BioMarin Pharmaceutical
BioMarin Pharmaceutical
Not Provided
Study Director: Debra Lounsbury BioMarin Pharmaceutical
BioMarin Pharmaceutical
July 2017

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