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Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01205152
First Posted: September 20, 2010
Last Update Posted: November 15, 2017
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):
Alexion Pharma GmbH
Results First Submitted: August 14, 2017  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Hypophosphatasia
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 main criteria for inclusion in Study ENB-002-08 were male and female patients </= 36 months of age, with severe infantile-onset HPP (symptom onset before 6 months of age) who were medically stable (ventilator support was allowed). To enter extension Study ENB-003-08, parent/guardian had to consent and patient had to complete Study ENB-002-08.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Asfotase Alfa An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week

Participant Flow:   Overall Study
    Asfotase Alfa
STARTED   10 
COMPLETED   9 
NOT COMPLETED   1 
Death                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.
Of the 11 patients who were enrolled in Study ENB-002-08, 10 patients completed Study ENB-002-08 and were enrolled in extension study ENB-003-08.

Reporting Groups
  Description
Asfotase Alfa An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week

Baseline Measures
   Asfotase Alfa 
Overall Participants Analyzed 
[Units: Participants]
 10 
Age [1] 
[Units: Weeks]
Mean (Standard Deviation)
 56.44  (61.888) 
[1] Mean (SD) age at time of first dose in ENB-002-08 (NCT00744042)
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      6  60.0% 
Male      4  40.0% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      1  10.0% 
Not Hispanic or Latino      9  90.0% 
Unknown or Not Reported      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of 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      10 100.0% 
More than one race      0   0.0% 
Unknown or Not Reported      0   0.0% 


  Outcome Measures
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1.  Primary:   Long-term Tolerability of Subcutaneous (SC) Asfotase Alfa   [ Time Frame: 84 months ]

2.  Primary:   Long-term Efficacy of Asfotase Alfa in Treating Rickets in Infants and Young Children With Hypophosphatasia (HPP).   [ Time Frame: Up to 90 Months ]

3.  Secondary:   Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Plasma Inorganic Pyrophosphate (PPi) Levels   [ Time Frame: Up to 90 Months ]

4.  Secondary:   Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Pyridoxal-5-phosphate (PLP) Levels   [ Time Frame: Up to 90 Months ]

5.  Secondary:   Effect of SC Asfotase Alfa on Growth: Weight Z-scores   [ Time Frame: Up to 90 Months ]

6.  Secondary:   Effect of SC Asfotase Alfa on Growth: Height/Length Z-scores   [ Time Frame: Up to 90 Months ]

7.  Secondary:   Effect of SC Asfotase Alfa on Respiratory Function   [ Time Frame: Up to 90 Months ]


  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: Director of Clinical Trials
Organization: Alexion Pharmaceuticals, Inc.
phone: 475-230-2596
e-mail: ClinicalTrials@alexion.com


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

Responsible Party: Alexion Pharma GmbH
ClinicalTrials.gov Identifier: NCT01205152     History of Changes
Other Study ID Numbers: ENB-003-08
FD-R-003745-03 ( Other Grant/Funding Number: Office of Orphan Product Development )
First Submitted: September 17, 2010
First Posted: September 20, 2010
Results First Submitted: August 14, 2017
Results First Posted: November 15, 2017
Last Update Posted: November 15, 2017