This site became the new ClinicalTrials.gov on June 19th. Learn more.
Show more
ClinicalTrials.gov Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu
Give us feedback

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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01415427
First received: August 8, 2011
Last updated: April 13, 2017
Last verified: April 2017
Results First Received: April 13, 2017  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Participant, Care Provider, Investigator, Outcomes Assessor;   Primary Purpose: Treatment
Conditions: Mucopolysaccharidosis IV A
Morquio A Syndrome
MPS IVA
Interventions: Drug: BMN 110 - Weekly
Drug: BMN 110 - Every Other Week

  Participant Flow
  Hide 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
PBO-QOW Weekly IV infusions of placebo solution for a total of 24 consecutive weeks in MOR004 + Biweekly infusions of 2.0 mg/kg/qow BMN 110 and infusions of placebo on alternating weeks in MOR005
PBO-QW Weekly IV infusions of placebo solution for a total of 24 consecutive weeks in MOR004 +Weekly infusions of 2.0 mg/kg/week BMN 110 in MOR005
QOW-QOW Biweekly infusions of 2.0 mg/kg/qow BMN 110 and infusions of placebo on alternating weeks for a total of 24 consecutive weeks in MOR004 + Biweekly infusions of 2.0 mg/kg/qow BMN 110 and infusions of placebo on alternating weeks in MOR005
QW-QW Weekly infusions of 2.0 mg/kg/week BMN 110 for a total of 24 consecutive weeks in MOR004 + Weekly infusions of 2.0 mg/kg/week BMN 110 in MOR005

Participant Flow:   Overall Study
    PBO-QOW   PBO-QW   QOW-QOW   QW-QW
STARTED   29   29   59   56 
COMPLETED   0   0   1   0 
NOT COMPLETED   29   29   58   56 



  Baseline Characteristics
  Hide 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
PBO-QOW Placebo in MOR004 + BMN110 2.0 mg/kg/qow in MOR005
PBO-QW Placebo in MOR004 + BMN110 2.0 mg/kg/qw in MOR005
QOW-QOW BMN110 2.0 mg/kg/qow in MOR004 + BMN110 2.0 mg/kg/qow in MOR005
QW-QW BMN110 2.0 mg/kg/qw in MOR004 + BMN110 2.0 mg/kg/qw in MOR005
Total Total of all reporting groups

Baseline Measures
   PBO-QOW   PBO-QW   QOW-QOW   QW-QW   Total 
Overall Participants Analyzed 
[Units: Participants]
 29   29   59   56   173 
Age 
[Units: Years]
Mean (Standard Deviation)
 16.7  (13.66)   13.5  (8.50)   15.3  (10.79)   12.8  (8.01)   14.4  (10.20) 
Age, Customized 
[Units: Participants]
         
5 - 11   15   15   31   32   93 
12 - 18   7   7   16   16   46 
>=19   7   7   12   8   34 
Sex: Female, Male 
[Units: Participants]
Count of Participants
         
Female      14  48.3%      18  62.1%      25  42.4%      30  53.6%      87  50.3% 
Male      15  51.7%      11  37.9%      34  57.6%      26  46.4%      86  49.7% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
         
Hispanic or Latino      4  13.8%      8  27.6%      16  27.1%      9  16.1%      37  21.4% 
Not Hispanic or Latino      25  86.2%      21  72.4%      43  72.9%      47  83.9%      136  78.6% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Race/Ethnicity, Customized 
[Units: Participants]
         
American Indian or Alaska Native   0   0   0   0   0 
Asian   4   7   15   14   40 
Native Hawaiian or Other Pacific Islander   0   0   0   0   0 
Black or African American   0   0   2   2   4 
White   25   18   35   35   113 
Other   0   4   7   5   16 
Region of Enrollment 
[Units: Participants]
         
Argentina   0   1   1   0   2 
Brazil   2   3   10   5   20 
Canada   3   1   5   5   14 
Colombia   0   2   2   2   6 
Denmark   0   0   0   1   1 
France   2   5   5   8   20 
Germany   2   2   5   1   10 
Italy   3   1   4   2   10 
Japan   0   0   4   2   6 
Korea, South   2   1   1   3   7 
Netherlands   1   0   2   3   6 
Portugal   1   1   1   0   3 
Qatar   0   1   0   1   2 
Saudi Arabia   0   2   1   4   7 
Taiwan   0   1   3   1   5 
United Kingdom   6   3   4   9   22 
United States   7   5   11   9   32 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Change From Baseline in 6-minute Walk (6MW) Test - ITT   [ Time Frame: Baseline to week 168 ]

2.  Primary:   Change From Baseline in 6-minute Walk (6MW) Test - MPP   [ Time Frame: Baseline to week 168 ]

3.  Secondary:   Change From Baseline in 3-minute Stair Climb Test - ITT   [ Time Frame: Baseline to week 168 ]

4.  Secondary:   Change From Baseline in 3-minute Stair Climb Test - MPP   [ Time Frame: Baseline to week 168 ]

5.  Secondary:   Change From Baseline in Urine Keratan Sulfate - ITT   [ Time Frame: Baseline to week 168 ]

6.  Secondary:   Change From Baseline in Urine Keratan Sulfate - MPP   [ Time Frame: Baseline to week 168 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide 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
  Hide 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
e-mail: pslasor@bmrn.com


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

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01415427     History of Changes
Other Study ID Numbers: MOR-005
Study First Received: August 8, 2011
Results First Received: April 13, 2017
Last Updated: April 13, 2017