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 GmbH
ClinicalTrials.gov Identifier:
NCT00744042
First received: August 27, 2008
Last updated: January 25, 2016
Last verified: January 2016
Results First Received: May 15, 2011  
Study Type: Interventional
Study Design: 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 patients received an initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa for the first week followed by regular administration of subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times/week (total 3 mg/kg/week).

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.
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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)
  1.11  (1.13)  
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:   Change in Rickets Severity From Baseline to Week 24, Based on Assessment of Skeletal Radiographs Using Radiologic Global Impression of Change (RGI-C)   [ Time Frame: 24 weeks ]

2.  Secondary:   Maximum Serum Concentration of Asfotase Alfa (Cmax)   [ Time Frame: Study Week 1 (0 to 168 hours post-dose). Study Week 2 and Study Week 3 (0 to 48 hours post-dose) ]

3.  Secondary:   Time at Maximum Serum Concentration of Asfotase Alfa (Tmax)   [ Time Frame: Study Week 1 (0 to 168 hours post-dose). Study Week 2 and Study Week 3 (0 to 48 hours post-dose). ]

4.  Secondary:   Area Under Serum Concentration-time Curve to Last Measurable Concentration of Asfotase Alfa (AUCt)   [ Time Frame: Study Week 1 (0 to 168 hours post-dose). Study Week 2 and Study Week 3 (0 to 48 hours post-dose). ]


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


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

Responsible Party: Alexion Pharma GmbH
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: January 25, 2016
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