A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT00884949
First received: April 10, 2009
Last updated: May 28, 2014
Last verified: May 2014
Results First Received: March 13, 2014  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: MPS IV A
Intervention: Drug: BMN 110

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
BMN 110

Dose-Escalation Period:

Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen:

  • Weeks 1-12: 0.1 mg/kg/week
  • Weeks 13-24: 1.0 mg/kg/week
  • Weeks 25-36: 2.0 mg/kg/week

Continuation Period:

Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week.


Participant Flow for 4 periods

Period 1:   Weeks 1-12: 0.1 mg/kg/Week
    BMN 110  
STARTED     20  
COMPLETED     18  
NOT COMPLETED     2  
Adverse Event                 1  
Withdrawal by Subject                 1  

Period 2:   Weeks 13-24: 1.0 mg/kg/Week
    BMN 110  
STARTED     18  
COMPLETED     18  
NOT COMPLETED     0  

Period 3:   Weeks 25-36: 2.0 mg/kg/Week
    BMN 110  
STARTED     18  
COMPLETED     18  
NOT COMPLETED     0  

Period 4:   Continuation Period
    BMN 110  
STARTED     18  
COMPLETED     18  
NOT COMPLETED     0  



  Baseline Characteristics
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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.
No text entered.

Reporting Groups
  Description
BMN 110 Dose-Escalation Period:

Baseline Measures
    BMN 110  
Number of Participants  
[units: participants]
  20  
Age  
[units: years]
Mean ± Standard Deviation
  8.0  ± 2.89  
Age, Customized  
[units: participants]
 
>=4 to <8 years     10  
>=8 to <10 years     6  
>=10 to <=18 years     4  
Gender  
[units: participants]
 
Female     8  
Male     12  
Ethnicity (NIH/OMB)  
[units: participants]
 
Hispanic or Latino     0  
Not Hispanic or Latino     20  
Unknown or Not Reported     0  
Race/Ethnicity, Customized  
[units: participants]
 
American Indian or Alaska Native     0  
Asian     9  
Native Hawaiian or Other Pacific Islander     0  
Black or African American     0  
White     9  
Other     2  
Region of Enrollment  
[units: participants]
 
United Kingdom     20  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Subject Incidence of Treatment Emergent AEs   [ Time Frame: Entire Study, through week 84 ]

2.  Secondary:   Change From Baseline in 6MWT   [ Time Frame: Baseline to Weeks 12, 24, 36, 48, 72 ]

3.  Secondary:   Change From Baseline in 3MSCT   [ Time Frame: Baseline to Weeks 12, 24, 36, 48, 72 ]

4.  Secondary:   Percent Change From Baseline in uKS   [ Time Frame: Baseline to Weeks 12, 24, 36, 72 ]

5.  Secondary:   Percent Change From Baseline in MVV   [ Time Frame: Baseline to Weeks 12, 24, 36, 72 ]

6.  Secondary:   Percent Change From Baseline in FVC   [ Time Frame: Baseline to Weeks 12, 24, 36, 72 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: BioMarin Medical Information Services
Organization: BioMarin Pharmaceutical Inc.
phone: 800-983-4587
e-mail: medinfo@bmrn.com


No publications provided


Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT00884949     History of Changes
Other Study ID Numbers: MOR-002
Study First Received: April 10, 2009
Results First Received: March 13, 2014
Last Updated: May 28, 2014
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
United Kingdom: Research Ethics Committee