Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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
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
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
Sex
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
1/03/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/09/2014
Sample size
Target
Accrual to date
Final
32
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, Inc.
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/study/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

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02104219