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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02104219
Registration number
NCT02104219
Ethics application status
Date submitted
25/03/2014
Date registered
4/04/2014
Date last updated
1/04/2019
Titles & IDs
Public title
Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
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Scientific title
A Retrospective, Non-interventional, Epidemiologic Study of the Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
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Secondary ID [1]
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ALX-HPP-502
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hypophosphatasia (HPP)
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Condition category
Condition code
Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Radiographic Global Impression of Change - RGI-C
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Assessment method [1]
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The RGI-C scale is a 7-point ordinal scale that is used to evaluate musculoskeletal characteristics of HPP (eg, metaphyseal fraying, demineralization of distal metaphyses). The scores range from -3 (severe worsening) to +3 (complete or near-complete healing).
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Timepoint [1]
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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.
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Secondary outcome [1]
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Change in Height Z-score From Baseline to Last Assessment
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Assessment method [1]
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Height measurements were assigned to Z-scores which were calculated using the Centers for Disease Control and Prevention (CDC) 2000 growth charts and methodology. The Z-score indicates the number of standard deviations away from the mean. 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.
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Timepoint [1]
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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.
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Secondary outcome [2]
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Change in Weight Z-score From Baseline to Last Assessment
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Assessment method [2]
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Weight measurements were assigned a Z-score which was calculated using the Centers for Disease Control and Prevention (CDC) 2000 growth charts and methodology. The Z-score indicates the number of standard deviations away from the mean. 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.
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Timepoint [2]
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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.
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Eligibility
Key inclusion criteria
- Documented informed consent unless patient is deceased
- Patients with Juvenile-onset HPP, defined as documented onset of first signs/symptoms
at = 6 months to ?18 years
- Documented diagnosis of HPP as indicated by skeletal manifestations and low alkaline
phosphatase or genotyping
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Minimum age
5
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Received treatment with asfotase alfa in the ENB-006-09 study and/or currently
enrolled in the ENB-008-10 study
- Received other treatment and/or intervention to treat HPP up to 15 years old
- Other clinically significant disease
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Study design
Purpose
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Duration
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Selection
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Timing
Retrospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/03/2014
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/09/2014
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Sample size
Target
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Accrual to date
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Final
32
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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- Sydney
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Recruitment postcode(s) [1]
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- Sydney
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Recruitment outside Australia
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United States of America
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Georgia
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Indiana
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Massachusetts
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Missouri
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North Carolina
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Ohio
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Texas
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Washington
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Country [9]
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Canada
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Manitoba
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Country [10]
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France
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State/province [10]
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Le Kremlin-Bicêtre
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France
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Paris
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France
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Toulouse
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Netherlands
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Rotterdam
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Country [14]
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Russian Federation
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State/province [14]
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Moscow
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Turkey
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Istanbul
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United Kingdom
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Manchester
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Country [17]
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United Kingdom
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State/province [17]
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Sheffield
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Alexion Pharmaceuticals, Inc.
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to characterize the natural history of HPP in patients with
Juvenile-onset HPP.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02104219
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Contact person for public queries
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02104219
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