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A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

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ClinicalTrials.gov Identifier: NCT01275066
Recruitment Status : Completed
First Posted : January 12, 2011
Results First Posted : April 15, 2014
Last Update Posted : July 7, 2014
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Brief Summary:

This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).

There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) may be a potential new treatment option for MPS IVA patients. BMN 110 is administered to MPS IVA patients by IV infusion, allowing cellular uptake by the mannose-6-phosphate receptor and transportation to the lysosomes.

This enzyme uptake into the lysosomes is hypothesized to promote increased catabolism of keratan sulfate (KS) in tissue macrophages, hyaline cartilage, other connective tissues, and heart valve, and reduce the progressive accumulation of KS which is responsible for the clinical manifestations of the disorders.

Condition or disease Intervention/treatment Phase
MPS IV A Drug: BMN 110 Weekly Drug: Placebo Drug: BMN 110 Every Other Week Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 177 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Study Start Date : February 2011
Actual Primary Completion Date : August 2012
Actual Study Completion Date : August 2012

Arm Intervention/treatment
Placebo Comparator: Placebo Drug: Placebo
Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week.

Experimental: BMN 110 Weekly Drug: BMN 110 Weekly
BMN 110 Weekly: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
Other Names:
  • recombinant human N-acetylgalactosamine-6-sulfatase
  • rhGALNS

Experimental: BMN 110 Every Other Week Drug: BMN 110 Every Other Week
BMN 110 Every Other Week: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks.
Other Names:
  • recombinant human N-acetylgalactosamine-6-sulfatase
  • rhGALNS

Primary Outcome Measures :
  1. Change From Baseline in Endurance as Measured by the 6-minute Walk Test [ Time Frame: Baseline to Week 24 ]

Secondary Outcome Measures :
  1. Change From Baseline in Endurance as Measured by the 3-minute Stair Climb Test [ Time Frame: Baseline to Week 24 ]
  2. Percent Change From Baseline in Urine Keratan Sulfate Normalized for Urine Creatinine [ Time Frame: Baseline to Week 24 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • At least 5 years of age.
  • 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.
  • Willing and able to provide written, signed informed consent, or in the case of patients under the age of 18 (or 16 years, depending on the region), provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
  • Must meet the study entrance requirements for the 6-minute walk test.
  • Sexually active patients must be willing to use an acceptable method of contraception while participating in the study.
  • Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study.

Exclusion Criteria:

  • Previous hematopoietic stem cell transplant (HSCT).
  • Previous treatment with BMN 110.
  • Has known hypersensitivity to any of the components of BMN 110.
  • Major surgery within 3 months prior to study entry or planned major surgery during the 24-week treatment period.
  • Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
  • 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.

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): NCT01275066

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United States, California
Oakland, California, United States
United States, Delaware
Wilmington, Delaware, United States
United States, District of Columbia
Washington, District of Columbia, United States
United States, Illinois
Chicago, Illinois, United States
United States, New York
New York, New York, United States
United States, Washington
Seattle, Washington, United States
Cordoba, Argentina
Campina Grande, Brazil
Porto Alegre, Brazil
Montreal, Canada
Sherbrooke, Canada
Toronto, Canada
Bogota, Colombia
Copenhagen, Denmark
Lyon, France
Paris, France
Mainz, Germany
Monza, Italy
Tokyo, Japan
Korea, Republic of
Seoul, Korea, Republic of
Amsterdam, Netherlands
Coimbra, Portugal
Doha, Qatar
Saudi Arabia
Riyadh, Saudi Arabia
Taipei, Taiwan
United Kingdom
Birmingham, United Kingdom
London, United Kingdom
Manchester, United Kingdom
Sponsors and Collaborators
BioMarin Pharmaceutical
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Study Director: Debra Lounsbury BioMarin Pharmaceutical
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01275066    
Other Study ID Numbers: MOR-004
2010-020198-18 ( EudraCT Number )
10/H1306/87 ( Other Identifier: National Research Ethics Service )
18972/0213/001-0001 ( Other Identifier: Medicines and Healthcare Products Regulatory Agency )
2011_038#B201129 ( Other Identifier: Medical Ethics Review Committee (METC) )
145240 ( Other Identifier: Health Canada )
2011-01-09 ( Other Identifier: Committee for the Protection of Personnes )
20110012889 ( Other Identifier: Korean Food & Drug Administration (KFDA) )
0999935174 ( Other Identifier: Taiwan FDA )
First Posted: January 12, 2011    Key Record Dates
Results First Posted: April 15, 2014
Last Update Posted: July 7, 2014
Last Verified: June 2014
Keywords provided by BioMarin Pharmaceutical:
Mucopolysaccharidosis IV type A
Mucopolysaccharidosis IVA
Morquio A Syndrome
Lysosomal Storage Disorder
N-acetylgalactosamine-6-sulfate sulfatase
enzyme replacement therapy
Additional relevant MeSH terms:
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Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases