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

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ClinicalTrials.gov Identifier: NCT01242111
Recruitment Status : Terminated
First Posted : November 16, 2010
Results First Posted : September 30, 2015
Last Update Posted : September 30, 2015
Sponsor:
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: MPS IV A
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
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 BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.

Participant Flow:   Overall Study
    BMN 110
STARTED   20 
COMPLETED   17 
NOT COMPLETED   3 
Adverse Event                1 
Withdrawal by Subject                1 
Did not consent to MOR-100 follow-up                1 



  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.
No text entered.

Reporting Groups
  Description
BMN 110 BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.

Baseline Measures
   BMN 110 
Overall Participants Analyzed 
[Units: Participants]
 20 
Age 
[Units: Years]
Mean (Standard Deviation)
 8.4  (2.90) 
Age, Customized 
[Units: Participants]
 
>=4 to < 8 years   10 
>=8 to <10 years   5 
>=10 to <=18 years   5 
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

1.  Primary:   Safety Evaluation   [ Time Frame: Entire Study Period, up to 240 weeks ]

2.  Secondary:   Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).   [ Time Frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial ]

3.  Secondary:   Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).   [ Time Frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial ]

4.  Secondary:   Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).   [ Time Frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 168 weeks during the MOR-100 extension trial ]

5.  Secondary:   Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).   [ Time Frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial ]

6.  Secondary:   Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).   [ Time Frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial ]


  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
No text entered.


  More Information

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


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01242111     History of Changes
Other Study ID Numbers: MOR-100
First Submitted: October 28, 2010
First Posted: November 16, 2010
Results First Submitted: July 10, 2015
Results First Posted: September 30, 2015
Last Update Posted: September 30, 2015