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

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Mucopolysaccharidosis IVA
Morquio A Syndrome
Intervention: Drug: BMN 110

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
BMN110 2.0 mg/kg/Week Weekly intravenous infusions of BMN 110 at a dose of 2.0 mg/kg for 52 consecutive weeks. Each infusion will be administered over a period of approximately 4 hours.

Participant Flow:   Overall Study
    BMN110 2.0 mg/kg/Week

  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
BMN110 2.0 mg/kg/Week 2.0 mg/kg/week

Baseline Measures
   BMN110 2.0 mg/kg/Week 
Overall Participants Analyzed 
[Units: Participants]
[Units: Years]
Mean (Standard Deviation)
 3.1  (1.34) 
Age, Customized 
[Units: Participants]
0 to <3 years   7 
>=3 to <5 years   8 
Sex: Female, Male 
[Units: Participants]
Count of Participants
Female      8  53.3% 
Male      7  46.7% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
Hispanic or Latino      1   6.7% 
Not Hispanic or Latino      14  93.3% 
Unknown or Not Reported      0   0.0% 
Race/Ethnicity, Customized 
[Units: Participants]
Asian   4 
White   10 
Other   1 
Region of Enrollment 
[Units: Participants]
Italy   2 
United Kingdom   6 
United States   7 
Urinary Keratan Sulfate Measures 
[Units: Ug/mg]
Mean (Standard Deviation)
 35.9  (12.32) 

  Outcome Measures

1.  Primary:   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 ]

2.  Secondary:   Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures   [ Time Frame: Baseline to Week 52 ]

3.  Secondary:   Change From Baseline in Normalized Growth Rate Z-Scores   [ Time Frame: Baseline to Week 52 ]

  Serious Adverse Events

  Other Adverse Events

  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact:  
Name/Title: Peter Slasor/Sr Director, Biostatistics, Global Clinical Sciences
Organization: BioMarin Pharmaceutical Inc.
phone: 415-506-6765

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
First Submitted: December 22, 2011
First Posted: January 24, 2012
Results First Submitted: April 13, 2017
Results First Posted: July 6, 2017
Last Update Posted: August 10, 2017