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Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

This study has been terminated.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01609062
First Posted: May 31, 2012
Last Update Posted: February 1, 2016
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.
Information provided by (Responsible Party):
BioMarin Pharmaceutical
Results First Submitted: November 12, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Mucopolysaccharidosis IVA
Morquio A Syndrome
MPS IVA
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 Weekly at 2.0 mg/kg/Week BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110 Weekly at 4.0 mg/kg/Week BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.

Participant Flow for 2 periods

Period 1:   Primary Phase
    BMN 110 Weekly at 2.0 mg/kg/Week   BMN 110 Weekly at 4.0 mg/kg/Week
STARTED   15   10 
COMPLETED   15   10 
NOT COMPLETED   0   0 

Period 2:   Extension Phase
    BMN 110 Weekly at 2.0 mg/kg/Week   BMN 110 Weekly at 4.0 mg/kg/Week
STARTED   15   10 
COMPLETED   0   0 [1] 
NOT COMPLETED   15   10 
Terminated by Sponsor                14                9 
Discontinued drug and then was pregnant                1                0 
Anticipated surgery & transition to EAP                0                1 
[1] EAP = Expanded Access Program



  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 Weekly at 2.0 mg/kg/Week BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110 Weekly at 4.0 mg/kg/Week BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Total Total of all reporting groups

Baseline Measures
   BMN 110 Weekly at 2.0 mg/kg/Week   BMN 110 Weekly at 4.0 mg/kg/Week   Total 
Overall Participants Analyzed 
[Units: Participants]
 15   10   25 
Age 
[Units: Years]
Mean (Standard Deviation)
 14.9  (9.32)   12.0  (3.16)   13.7  (7.52) 
Age, Customized 
[Units: Participants]
     
7 to 11 years   9   5   14 
12 to 18 years   3   5   8 
>=19 years   3   0   3 
Gender 
[Units: Participants]
     
Female   12   4   16 
Male   3   6   9 
Ethnicity (NIH/OMB) 
[Units: Participants]
     
Hispanic or Latino   4   2   6 
Not Hispanic or Latino   11   8   19 
Unknown or Not Reported   0   0   0 
Race/Ethnicity, Customized 
[Units: Participants]
     
American Indian or Alaska Native   0   0   0 
Asian   0   0   0 
Native Hawaiian or Other Pacific Islander   0   0   0 
Black or African American   0   0   0 
White   14   9   23 
Other   1   1   2 
Region of Enrollment 
[Units: Participants]
     
Canada   5   1   6 
Germany   0   3   3 
United Kingdom   1   1   2 
United States   9   5   14 


  Outcome Measures
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1.  Primary:   Safety Evaluation   [ Time Frame: Entire Study Period, up to 192 weeks or ETV (early termination visit) ]

2.  Secondary:   6-minute Walk Test (6MWT)   [ Time Frame: Baseline, Week 12, 24, and 52 ]

3.  Secondary:   3-minute Stair Climb Test (3MSCT)   [ Time Frame: Baseline, Week 12, 24, and 52 ]

4.  Secondary:   Respiratory Function Test (MVV and FVC)   [ Time Frame: Baseline, Week 12, 24, and 52 ]

5.  Secondary:   Normalized Urine Keratan Sulfate (uKS)   [ Time Frame: Baseline, Week 12, 24, and 52 ]

6.  Secondary:   Cardiopulmonary Exercise Testing (CPET) - Duration of Exercise   [ Time Frame: Baseline, Week 25 and 52 ]

7.  Secondary:   Cardiopulmonary Exercise Testing (CPET) - Peak Workload   [ Time Frame: Baseline, Week 25 and 52 ]

8.  Secondary:   Cardiopulmonary Exercise Testing (CPET) - O2 Pulse   [ Time Frame: Baseline, Week 25 and 52 ]

9.  Secondary:   Cardiopulmonary Exercise Testing (CPET) - Aerobic Efficiency   [ Time Frame: Baseline, Week 25 and 52 ]

10.  Secondary:   Muscle Strength Testing (MST) - Knee Extension Test   [ Time Frame: Baseline, Week 25 and 52 ]

11.  Secondary:   Muscle Strength Testing (MST) - Knee Flexion Test   [ Time Frame: Baseline, Week 25 and 52 ]

12.  Secondary:   Muscle Strength Testing (MST) - Elbow Flexion Test   [ Time Frame: Baseline, Week 25 and 52 ]

13.  Secondary:   Adolescent Pediatric Pain Tool (APPT) - Pain Intensity   [ Time Frame: Baseline, Week 12, 24, and 52 ]


  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



Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01609062     History of Changes
Other Study ID Numbers: MOR-008
First Submitted: May 24, 2012
First Posted: May 31, 2012
Results First Submitted: November 12, 2015
Results First Posted: February 1, 2016
Last Update Posted: February 1, 2016