Trial Review

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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
19/09/2024

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
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Missouri
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
North Carolina
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Tennessee
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
United States of America
State/province [16] 0 0
Wisconsin
Country [17] 0 0
Canada
State/province [17] 0 0
Manitoba
Country [18] 0 0
France
State/province [18] 0 0
Haute Garonne
Country [19] 0 0
France
State/province [19] 0 0
Paris
Country [20] 0 0
France
State/province [20] 0 0
Lyon
Country [21] 0 0
France
State/province [21] 0 0
Île-de-France
Country [22] 0 0
Germany
State/province [22] 0 0
Bayern
Country [23] 0 0
Germany
State/province [23] 0 0
Nordrhein Westfalen
Country [24] 0 0
Germany
State/province [24] 0 0
Sachsen
Country [25] 0 0
Germany
State/province [25] 0 0
Berlin
Country [26] 0 0
Germany
State/province [26] 0 0
Giessen
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Germany
State/province [27] 0 0
Hamburg
Country [28] 0 0
Germany
State/province [28] 0 0
Homburg
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Germany
State/province [29] 0 0
Koeln
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Germany
State/province [30] 0 0
Mainz
Country [31] 0 0
Germany
State/province [31] 0 0
Muenchen
Country [32] 0 0
Germany
State/province [32] 0 0
Müllheim
Country [33] 0 0
Germany
State/province [33] 0 0
Rostock
Country [34] 0 0
Italy
State/province [34] 0 0
Firenze
Country [35] 0 0
Italy
State/province [35] 0 0
Pisa
Country [36] 0 0
Italy
State/province [36] 0 0
Rome
Country [37] 0 0
Italy
State/province [37] 0 0
San Giovanni Rotondo
Country [38] 0 0
Italy
State/province [38] 0 0
Verona
Country [39] 0 0
Poland
State/province [39] 0 0
Warsaw
Country [40] 0 0
Russian Federation
State/province [40] 0 0
Moscow
Country [41] 0 0
Russian Federation
State/province [41] 0 0
Saint Petersburg
Country [42] 0 0
Saudi Arabia
State/province [42] 0 0
Riyadh
Country [43] 0 0
Spain
State/province [43] 0 0
Barcelona
Country [44] 0 0
Spain
State/province [44] 0 0
Madrid
Country [45] 0 0
United Kingdom
State/province [45] 0 0
Avon
Country [46] 0 0
United Kingdom
State/province [46] 0 0
Belfast
Country [47] 0 0
United Kingdom
State/province [47] 0 0
Birmingham
Country [48] 0 0
United Kingdom
State/province [48] 0 0
Dundee
Country [49] 0 0
United Kingdom
State/province [49] 0 0
Leicester
Country [50] 0 0
United Kingdom
State/province [50] 0 0
London
Country [51] 0 0
United Kingdom
State/province [51] 0 0
Newcastle upon Tyne
Country [52] 0 0
United Kingdom
State/province [52] 0 0
Newport
Country [53] 0 0
United Kingdom
State/province [53] 0 0
Norwich
Country [54] 0 0
United Kingdom
State/province [54] 0 0
Oxford
Country [55] 0 0
United Kingdom
State/province [55] 0 0
Sheffield
Country [56] 0 0
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