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Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharma GmbH
ClinicalTrials.gov Identifier:
NCT01203826
First received: September 15, 2010
Last updated: June 27, 2017
Last verified: June 2017
Results First Received: June 27, 2017  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (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 main criteria for inclusion in Study ENB-006-09 were patients ages 5 to 12 years inclusive, with open growth plates at time of study entry and a documented diagnosis of HPP. To enter the extension, Study ENB-008-10, patients had to successfully complete Study ENB-006-09 and provide consent.

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
2 mg/kg Asfotase Alfa The starting dose of asfotase alfa was 3 mg/kg/week for all patients in Study ENB-008-10 and was subsequently increased per study-wide dose adjustment to a total dose of 6 mg/kg/week. Results are shown by the patient's dose group assignment from Study ENB-006-09.
3 mg/kg Asfotase Alfa The starting dose of asfotase alfa was 3 mg/kg/week for all patients in Study ENB-008-10 and was subsequently increased per study-wide dose adjustment to a total dose of 6 mg/kg/week. Results are shown by the patient's dose group assignment from Study ENB-006-09.

Participant Flow:   Overall Study
    2 mg/kg Asfotase Alfa   3 mg/kg Asfotase Alfa
STARTED   6 [1]   6 [1] 
COMPLETED   6   6 
NOT COMPLETED   0   0 
[1] Dose cohorts are per assigned dose in ENB-006-09. Results presented are for Study ENB-008-10 only.



  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
2 mg/kg Asfotase Alfa Dose group shown is as per patient's randomization in Study ENB-006-09
3 mg/kg Asfotase Alfa Dose group shown is as per patient's randomization in Study ENB-006-09
Total Total of all reporting groups

Baseline Measures
   2 mg/kg Asfotase Alfa   3 mg/kg Asfotase Alfa   Total 
Overall Participants Analyzed 
[Units: Participants]
 6   6   12 
Age [1] 
[Units: Years]
Mean (Standard Deviation)
 8.4  (2.21)   9.0  (2.51)   8.7  (2.27) 
[1] Age at Enrollment
Age, Customized [1] 
[Units: Participants]
Count of Participants
     
Age Group at Enrollment: 2 to 11 Years; %   5   5   10 
Age Group at Enrollment: 12 to 17 Years; %   1   1   2 
[1] n %
Sex: Female, Male [1] 
[Units: Participants]
Count of Participants
     
Female      1  16.7%      1  16.7%      2  16.7% 
Male      5  83.3%      5  83.3%      10  83.3% 
[1] n %
Ethnicity (NIH/OMB) [1] 
[Units: Participants]
Count of Participants
     
Hispanic or Latino      1  16.7%      0   0.0%      1   8.3% 
Not Hispanic or Latino      5  83.3%      6 100.0%      11  91.7% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
[1] n %
Race (NIH/OMB) [1] 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      0   0.0%      0   0.0%      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      0   0.0%      0   0.0%      0   0.0% 
White      6 100.0%      6 100.0%      12 100.0% 
More than one race      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
[1] n %
Region of Enrollment 
[Units: Participants]
Count of Participants
     
Canada   2   1   3 
United States   4   5   9 
Hypophosphatasia Phenotype [1] 
[Units: Participants]
Count of Participants
     
Infantile (< 6 months)      3  50.0%      1  16.7%      4  33.3% 
Juvenile (≥ 6 months to < 18 yrs)      3  50.0%      5  83.3%      8  66.7% 
[1] n %
Age at Onset of Hypophosphatasia Symptoms 
[Units: Months]
Mean (Standard Deviation)
 10.8  (8.66)   11.5  (5.54)   11.2  (6.94) 
Tanner Stage 1 [1] 
[Units: Participants]
Count of Participants
 6   6   12 
[1] n %


  Outcome Measures

1.  Primary:   Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09.   [ Time Frame: At least 72 months of treatment with asfotase alfa ]


  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: NCT01203826     History of Changes
Other Study ID Numbers: ENB-008-10
Study First Received: September 15, 2010
Results First Received: June 27, 2017
Last Updated: June 27, 2017