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

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
BioMarin Pharmaceutical Identifier:
First received: December 22, 2011
Last updated: September 10, 2014
Last verified: September 2014
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 Intervention Phase
Mucopolysaccharidosis IVA
Morquio A Syndrome
Drug: BMN 110
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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)

Resource links provided by NLM:

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • Descriptive summary of clinical safety assessments [ Time Frame: Up to 208 weeks/ETV ] [ Designated as safety issue: Yes ]

    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.

Secondary Outcome Measures:
  • Change in Urinary Keratan Sulfate measures over time [ Time Frame: Baseline, and weeks: 2, 4, 8, 13, 26, 39, 52, 78, 104, 130, 156, 182, 208/ETV ] [ Designated as safety issue: No ]
  • Change in patient growth over time [ Time Frame: Baseline and Weeks: 13, 26, 39, 52,78, 104, 130, 156, 182, 208/ETV ] [ Designated as safety issue: No ]
    Changes in growth over time will be assessed using anthropometric measurements and radiographs of lower extremities.

Estimated Enrollment: 15
Study Start Date: October 2011
Estimated Study Completion Date: June 2015
Estimated Primary Completion Date: June 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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


Ages Eligible for Study:   up to 5 Years
Genders Eligible for Study:   Both
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 patient at high risk of poor treatment compliance or of not completing the study.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01515956

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
Study Director: Adam Shaywitz, MD, PhD BioMarin Pharmaceutical
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: BioMarin Pharmaceutical Identifier: NCT01515956     History of Changes
Other Study ID Numbers: MOR-007 
Study First Received: December 22, 2011
Last Updated: September 10, 2014
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United Kingdom: National Institute for Health Research
United Kingdom: Research Ethics Committee
Italy: The Italian Medicines Agency
Italy: Ethics Committee
Taiwan : Food and Drug Administration
Taiwan: Department of Health

Keywords provided by BioMarin Pharmaceutical:
Mucopolysaccharidosis IVA type A
Mucopolysaccharidosis IVA
Morquio A Syndrome
Lysosomal Storage Disorder
enzyme replacement therapy

Additional relevant MeSH terms:
Mucopolysaccharidosis IV
Carbohydrate Metabolism, Inborn Errors
Connective Tissue Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Pathologic Processes processed this record on May 26, 2016