Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharma International Sarl
ClinicalTrials.gov Identifier:
NCT00744042
First received: August 27, 2008
Last updated: May 27, 2014
Last verified: May 2014
Results First Received: May 15, 2011  
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: Hypophosphatasia (HPP)
Intervention: Biological: asfotase alfa

  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
The trial was posted on clinicaltrials.gov. Physicians managing the care of infants and young children with a confirmed diagnosis of HPP contacted existing sites or requested assistance with site set up from the sponsor.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
All screened patients met eligibility criteria and were enrolled in the study.

Reporting Groups
  Description
Asfotase Alfa All enrolled patients receive a single IV (intravenous) dose of Asfotase Alfa of 2 mg/kg followed by 7 days of observation. Following an assessment of safety data by an independent Data Safety Monitoring Board (DSMB), patients begin thrice weekly SC (subcutaneous) injections of Asfotase Alfa at a dose of 1 mg/kg for the remaining 23 weeks of the study.

Participant Flow:   Overall Study
    Asfotase Alfa  
STARTED     11  
COMPLETED     10  
NOT COMPLETED     1  
Withdrawal by Subject                 1  



  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
Asfotase Alfa All enrolled patients receive a single IV (intravenous) dose of Asfotase Alfa of 2 mg/kg followed by 7 days of observation. Following an assessment of safety data by an independent Data Safety Monitoring Board (DSMB), patients begin thrice weekly SC (subcutaneous) injections of Asfotase Alfa at a dose of 1 mg/kg for the remaining 23 weeks of the study.

Baseline Measures
    Asfotase Alfa  
Number of Participants  
[units: participants]
  11  
Age  
[units: participants]
 
<=18 years     11  
Between 18 and 65 years     0  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  1.06  ± 1.12  
Gender  
[units: participants]
 
Female     7  
Male     4  
Region of Enrollment  
[units: participants]
 
Canada     1  
United States     7  
United Arab Emirates     1  
United Kingdom     2  



  Outcome Measures
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1.  Primary:   Number of Patients Showing Radiographic Response After 24 Weeks of Treatment   [ Time Frame: 24 weeks ]

2.  Primary:   Number of SAEs (Serious Adverse Events) for All Treated Patients   [ Time Frame: 24 weeks ]


  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 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.
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: Alexion Pharma International Sàrl
Organization: Alexion Pharma International Sàrl
e-mail: ClinicalTrials@alxn.com


Publications:
Publications automatically indexed to this study:

Responsible Party: Alexion Pharma International Sarl
ClinicalTrials.gov Identifier: NCT00744042     History of Changes
Other Study ID Numbers: ENB-002-08
Study First Received: August 27, 2008
Results First Received: May 15, 2011
Last Updated: May 27, 2014
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United Arab Emirates: General Authority for Health Services for Abu Dhabi