Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

This study has been terminated.
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01697319
First received: September 17, 2012
Last updated: December 8, 2015
Last verified: December 2015
Results First Received: October 12, 2015  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Mucopolysaccharidosis IVA
Morquio A Syndrome
MPS IVA
Intervention: Drug: BMN 110

  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
BMN 110 at 2.0 mg/kg/Week Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for an initial treatment phase of 48 weeks and an extension treatment phase of up to an additional 96 weeks.

Participant Flow for 2 periods

Period 1:   Primary Phase
    BMN 110 at 2.0 mg/kg/Week
STARTED   16 [1] 
COMPLETED   15 
NOT COMPLETED   1 
Withdrawal by Subject                1 
[1] One subject enrolled but did not receive a single dose of study drug

Period 2:   Extension Phase
    BMN 110 at 2.0 mg/kg/Week
STARTED   15 
COMPLETED   0 
NOT COMPLETED   15 
Terminated by Sponsor                15 



  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
BMN 110 at 2.0 mg/kg/Week Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for an initial treatment phase of 48 weeks and an extension treatment phase of up to an additional 96 weeks.

Baseline Measures
   BMN 110 at 2.0 mg/kg/Week 
Overall Participants Analyzed 
[Units: Participants]
 16 
Age 
[Units: Years]
Mean (Standard Deviation)
 20.7  (8.38) 
Age, Customized 
[Units: Participants]
 
5 to 11 years   1 
12 to 18 years   8 
>=19 years   7 
Gender 
[Units: Participants]
 
Female   7 
Male   9 
Ethnicity (NIH/OMB) 
[Units: Participants]
 
Hispanic or Latino   4 
Not Hispanic or Latino   12 
Unknown or Not Reported   0 
Race/Ethnicity, Customized 
[Units: Participants]
 
American Indian or Alaska Native   0 
Asian   2 
Native Hawaiian or Other Pacific Islander   0 
Black or African American   1 
White   12 
Other   1 
Region of Enrollment 
[Units: Participants]
 
Germany   6 
United Kingdom   4 
United States   6 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)   [ Time Frame: Up to 96 weeks ]

2.  Primary:   Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)   [ Time Frame: Up to 96 weeks ]

3.  Primary:   Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT)   [ Time Frame: Up to 96 weeks ]

4.  Secondary:   Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS)   [ Time Frame: Up to 96 weeks ]


  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:  
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 Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


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: NCT01697319     History of Changes
Other Study ID Numbers: MOR-006
2011-005703-33 ( EudraCT Number )
Study First Received: September 17, 2012
Results First Received: October 12, 2015
Last Updated: December 8, 2015
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration
Germany: Federal Institute for Drugs and Medical Devices
Germany: Ethics Commission
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United Kingdom: Research Ethics Committee