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Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)

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ClinicalTrials.gov Identifier: NCT02104219
Recruitment Status : Completed
First Posted : April 4, 2014
Results First Posted : December 18, 2015
Last Update Posted : December 18, 2015
Sponsor:
Information provided by (Responsible Party):
Alexion Pharma GmbH

Study Type: Observational
Study Design: Time Perspective: Retrospective
Condition: Hypophosphatasia (HPP)

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Patients were enrolled between March 2014 and September 2014. Fifty-four patients were screened and 32 patients were enrolled at 15 centers across the United States, Canada, Australia, Turkey, The Netherlands, and the United Kingdom.

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
Retrospective Observational Patients diagnosed with juvenile-onset HPP (ie, documented onset of first signs/symptoms of HPP at ≥ 6 months to < 18 years of age).

Participant Flow:   Overall Study
    Retrospective Observational
STARTED   32 
COMPLETED   32 
NOT COMPLETED   0 



  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
Patients Diagnosed With Juvenile-onset HPP Patients diagnosed with juvenile-onset HPP (ie, documented onset of first signs/symptoms of HPP at ≥ 6 months to < 18 years of age).

Baseline Measures
   Patients Diagnosed With Juvenile-onset HPP 
Overall Participants Analyzed 
[Units: Participants]
 32 
Age, Customized [1] 
[Units: Months]
Mean (Standard Deviation)
 47  (32.21) 
[1] Age at diagnosis
Gender 
[Units: Participants]
 
Female   10 
Male   22 
Race/Ethnicity, Customized 
[Units: Participants]
 
White   31 
Unknown or Not Reported   1 
Region of Enrollment 
[Units: Participants]
 
Australia   2 
North America   25 
Europe   5 
Rickets Severity Scale - RSS [1] 
[Units: Units on a scale]
Median (Inter-Quartile Range)
 1.00 
 (0.00 to 1.50) 
[1] The RSS is a 10-point scale, developed for nutritional rickets, that evaluates the degree of metaphyseal cupping and fraying and the proportion of growth plate affected (10 points = severe cupping/fraying, 0 points = absence of cupping/fraying).
Growth Measured by Height Z-score [1] 
[Units: Z-score]
Median (Inter-Quartile Range)
 -0.86 
 (-1.57 to -0.62) 
[1] The Z-score indicates the number of standard deviations away from the mean 0. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome.
Growth Measured by Weight Z-score [1] 
[Units: Z-score]
Median (Inter-Quartile Range)
 -0.86 
 (-1.45 to -0.49) 
[1] The Z-score indicates the number of standard deviations away from the mean 0. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome.


  Outcome Measures

1.  Primary:   Radiographic Global Impression of Change - RGI-C   [ Time Frame: Between Baseline (earliest available, complete, and readable x-ray set) and all available, readable post-Baseline x-ray sets during the period of patients' aged 5 to 15 years, inclusive. ]

2.  Secondary:   Change in Height Z-score From Baseline to Last Assessment   [ Time Frame: Any available growth data during the period of patients’ aged 5 to 15 years, inclusive. Baseline is the earliest available assessment while post baselines are time points after Baseline during the defined age period. ]

3.  Secondary:   Change in Weight Z-score From Baseline to Last Assessment   [ Time Frame: Any available growth data during the period of patients’ aged 5 to 15 years, inclusive. Baseline is the earliest available assessment while Post baselines are time points after Baseline during the defined age period. ]

4.  Other Pre-specified:   Rickets Severity Sale - RSS   [ Time Frame: Any available data during the period of patients’ aged 5 to 15 years, inclusive. Baseline is the earliest available assessment value during the period. ]


  Serious Adverse Events


  Other Adverse Events


  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

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 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.


Results Point of Contact:  
Name/Title: Senior Medical Director, Clinical Development
Organization: Alexion Pharmaceuticals
phone: 203-272-2596
e-mail: clinicaltrials@alxn.com



Responsible Party: Alexion Pharma GmbH
ClinicalTrials.gov Identifier: NCT02104219     History of Changes
Obsolete Identifiers: NCT02104206
Other Study ID Numbers: ALX-HPP-502
First Submitted: March 25, 2014
First Posted: April 4, 2014
Results First Submitted: August 25, 2015
Results First Posted: December 18, 2015
Last Update Posted: December 18, 2015