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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02306720




Registration number
NCT02306720
Ethics application status
Date submitted
26/11/2014
Date registered
3/12/2014
Date last updated
7/05/2024

Titles & IDs
Public title
Registry of Patients With Hypophosphatasia
Scientific title
An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)
Secondary ID [1] 0 0
ALX-HPP-501
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]
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
Natural History Information
Timepoint [1] 0 0
1 Year
Primary outcome [2] 0 0
Burden of Disease/Patient-reported Outcomes
Timepoint [2] 0 0
1 year
Primary outcome [3] 0 0
Characterize the epidemiology of the HPP population.
Timepoint [3] 0 0
1 year
Primary outcome [4] 0 0
Long-Term Safety and Effectiveness of Asfotase Alfa
Timepoint [4] 0 0
1 year

Eligibility
Key inclusion criteria
* Male and female participants, of any age, with a confirmed diagnosis of HPP.
* Participant must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation.
* Participant or legal representative is able to read and/or understand the informed consent and study questionnaires in the local language.
* Signed informed consent and medical records release by the patient or legal representative. Patient or patient's parent/legal representative must be willing and able to give written informed consent, and the patient must be willing to give written informed assent, if appropriate and required by local regulations.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Currently participating in an Alexion-sponsored clinical trial. Enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
2/04/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/12/2031
Actual
Sample size
Target
900
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Research Site - Westmead
Recruitment hospital [2] 0 0
Research Site - Clayton
Recruitment hospital [3] 0 0
Research Site - Parkville
Recruitment hospital [4] 0 0
Research Site - Herston
Recruitment hospital [5] 0 0
Research Site - South Brisbane
Recruitment postcode(s) [1] 0 0
- Westmead
Recruitment postcode(s) [2] 0 0
- Clayton
Recruitment postcode(s) [3] 0 0
- Parkville
Recruitment postcode(s) [4] 0 0
- Herston
Recruitment postcode(s) [5] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
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United States of America
State/province [2] 0 0
Colorado
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United States of America
State/province [3] 0 0
Connecticut
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United States of America
State/province [4] 0 0
Florida
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United States of America
State/province [5] 0 0
Georgia
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United States of America
State/province [6] 0 0
Illinois
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United States of America
State/province [7] 0 0
Maryland
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United States of America
State/province [8] 0 0
Massachusetts
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United States of America
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Missouri
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United States of America
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New York
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United States of America
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North Carolina
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United States of America
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Ohio
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United States of America
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Pennsylvania
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United States of America
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Tennessee
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United States of America
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Utah
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United States of America
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Wisconsin
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Canada
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Manitoba
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France
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Haute Garonne
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France
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Paris
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France
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Lyon
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France
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Île-de-France
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Germany
State/province [22] 0 0
Bayern
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Germany
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Nordrhein Westfalen
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Germany
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Sachsen
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Germany
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Berlin
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Germany
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Giessen
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Germany
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Hamburg
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Germany
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Homburg
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Germany
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Koeln
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Germany
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Mainz
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Germany
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Muenchen
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Germany
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Müllheim
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Germany
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Rostock
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Italy
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Firenze
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Italy
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Pisa
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Italy
State/province [36] 0 0
Rome
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Italy
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San Giovanni Rotondo
Country [38] 0 0
Italy
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Verona
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Poland
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Warsaw
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Russian Federation
State/province [40] 0 0
Moscow
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Russian Federation
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Saint Petersburg
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Saudi Arabia
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Riyadh
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Spain
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Barcelona
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Spain
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Madrid
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United Kingdom
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Avon
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United Kingdom
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Belfast
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United Kingdom
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Birmingham
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United Kingdom
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Dundee
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United Kingdom
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Leicester
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United Kingdom
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London
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United Kingdom
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Newcastle upon Tyne
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United Kingdom
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Newport
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United Kingdom
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Norwich
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United Kingdom
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Oxford
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United Kingdom
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Sheffield
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United Kingdom
State/province [56] 0 0
Stanmore

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT02306720