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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02306720
Ethics application status
Date submitted
26/11/2014
Date registered
3/12/2014
Date last updated
17/09/2019

Titles & IDs
Public title
An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)
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
Patient-reported Outcomes - Age-appropriate PRO data will be collected using instruments to asses below:
Pain
Timepoint [1] 0 0
1 year
Primary outcome [2] 0 0
Patient-reported Outcomes - Age-appropriate PRO data will be collected using instruments to asses below:
Motor Capacity
Timepoint [2] 0 0
1 year
Primary outcome [3] 0 0
Patient-reported Outcomes - Age-appropriate PRO data will be collected using instruments to asses below:
Functional Status/Disability, including Activities of Daily Living (ADL)
Timepoint [3] 0 0
1 year
Primary outcome [4] 0 0
Patient-reported Outcomes - Age-appropriate PRO data will be collected using instruments to asses below:
Quality of Life (QoL)
Timepoint [4] 0 0
1 year

Eligibility
Key inclusion criteria
Male and female patients, of any age, with a confirmed diagnosis of HPP. Patient 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
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Currently participating in an Alexion-sponsored clinical trial or previously received
asfotase alfa. 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
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)
NSW,VIC
Recruitment hospital [1] 0 0
- Westmead
Recruitment hospital [2] 0 0
- Parkville
Recruitment postcode(s) [1] 0 0
- Westmead
Recruitment postcode(s) [2] 0 0
- Parkville
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
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
Rhode Island
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
Canada
State/province [16] 0 0
Manitoba
Country [17] 0 0
France
State/province [17] 0 0
Haute Garonne
Country [18] 0 0
France
State/province [18] 0 0
Paris
Country [19] 0 0
France
State/province [19] 0 0
Île-de-France
Country [20] 0 0
Germany
State/province [20] 0 0
Bayern
Country [21] 0 0
Germany
State/province [21] 0 0
Nordrhein-Westfalen
Country [22] 0 0
Germany
State/province [22] 0 0
Sachsen
Country [23] 0 0
Ireland
State/province [23] 0 0
Dublin
Country [24] 0 0
Italy
State/province [24] 0 0
Firenze
Country [25] 0 0
Italy
State/province [25] 0 0
Genova
Country [26] 0 0
Italy
State/province [26] 0 0
Milano
Country [27] 0 0
Italy
State/province [27] 0 0
Pisa
Country [28] 0 0
Italy
State/province [28] 0 0
Rome
Country [29] 0 0
Italy
State/province [29] 0 0
San Giovanni Rotondo
Country [30] 0 0
Italy
State/province [30] 0 0
Verona
Country [31] 0 0
Portugal
State/province [31] 0 0
Lisboa
Country [32] 0 0
Russian Federation
State/province [32] 0 0
Kurgan
Country [33] 0 0
Russian Federation
State/province [33] 0 0
Moscow
Country [34] 0 0
Russian Federation
State/province [34] 0 0
Saint Petersburg
Country [35] 0 0
Saudi Arabia
State/province [35] 0 0
Riyadh
Country [36] 0 0
Spain
State/province [36] 0 0
Barcelona
Country [37] 0 0
Spain
State/province [37] 0 0
Madrid
Country [38] 0 0
Spain
State/province [38] 0 0
Móstoles
Country [39] 0 0
Spain
State/province [39] 0 0
Palma
Country [40] 0 0
Spain
State/province [40] 0 0
Pamplona
Country [41] 0 0
United Kingdom
State/province [41] 0 0
Avon
Country [42] 0 0
United Kingdom
State/province [42] 0 0
Birmingham
Country [43] 0 0
United Kingdom
State/province [43] 0 0
Leicester
Country [44] 0 0
United Kingdom
State/province [44] 0 0
London
Country [45] 0 0
United Kingdom
State/province [45] 0 0
Manchester
Country [46] 0 0
United Kingdom
State/province [46] 0 0
Newcastle upon Tyne
Country [47] 0 0
United Kingdom
State/province [47] 0 0
Norwich
Country [48] 0 0
United Kingdom
State/province [48] 0 0
Sheffield
Country [49] 0 0
United Kingdom
State/province [49] 0 0
Stanmore

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

Ethics approval
Ethics application status

Summary
Brief summary
To collect and evaluate long-term safety and effectiveness data in HPP patients who have/ are
receiving treatment with asfotase alfa.
Trial website
https://clinicaltrials.gov/show/NCT02306720
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/show/NCT02306720