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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/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)
Scientific title
A Retrospective, Non-interventional, Epidemiologic Study of the Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
Secondary ID [1] 0 0
ALX-HPP-502
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypophosphatasia (HPP) 0 0
Condition category
Condition code

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Radiographic Global Impression of Change - RGI-C - 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).
Timepoint [1] 0 0
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.
Secondary outcome [1] 0 0
Change in Height Z-score From Baseline to Last Assessment - 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.
Timepoint [1] 0 0
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.
Secondary outcome [2] 0 0
Change in Weight Z-score From Baseline to Last Assessment - 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.
Timepoint [2] 0 0
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.

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
Minimum age
5 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose
Duration
Selection
Timing
Retrospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Sydney
Recruitment postcode(s) [1] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia
Country [2] 0 0
United States of America
State/province [2] 0 0
Indiana
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
North Carolina
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Washington
Country [9] 0 0
Canada
State/province [9] 0 0
Manitoba
Country [10] 0 0
France
State/province [10] 0 0
Le Kremlin-Bicêtre
Country [11] 0 0
France
State/province [11] 0 0
Paris
Country [12] 0 0
France
State/province [12] 0 0
Toulouse
Country [13] 0 0
Netherlands
State/province [13] 0 0
Rotterdam
Country [14] 0 0
Russian Federation
State/province [14] 0 0
Moscow
Country [15] 0 0
Turkey
State/province [15] 0 0
Istanbul
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Manchester
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Alexion Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to characterize the natural history of HPP in patients with
Juvenile-onset HPP.
Trial website
https://clinicaltrials.gov/show/NCT02104219
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications