Registry of Patients With Hypophosphatasia

Hypophosphatasia (HPP) Clinical Trial

Official title:

An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT02306720
• Other study ID #: ALX-HPP-501
• Status: Enrolling by invitation
• Start date: December 31, 2014
• Est. completion date: August 28, 2030

Study information

• Verified date: July 2023
• Source: Alexion
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

In this prospective, observational, long term registry patients of all ages with a diagnosis of hypophosphatasia (HPP) are followed at participating sites in multiple countries.

Description:

The HPP Registry is an observational, prospective, long-term registry designed to collect data on HPP epidemiology, disease history, clinical course, symptoms and burden of disease from patients of all ages who have a diagnosis of HPP. Evaluation of safety and effectiveness data in patients with HPP who have/are receiving treatment with Asfotase alfa

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 900
• Est. completion date: August 28, 2030
• Est. primary completion date: December 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

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.

Related Conditions & MeSH terms

• Hypophosphatasia
• Hypophosphatasia (HPP)

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Alexion

Countries where clinical trial is conducted

United States,  Australia,  Canada,  France,  Germany,  Italy,  Poland,  Russian Federation,  Saudi Arabia,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Natural History Information	To collect information on the natural history of HPP from patients of all ages, including pediatric patients and adults with HPP, regardless of age at onset.	1 Year
Primary	Burden of Disease/Patient-reported Outcomes	Age-appropriate PRO data will be collected using instruments to asses below: Pain, Motor Capacity, Functional Status/Disability, including Activities of Daily Living (ADL), Quality of Life (QoL)	1 year
Primary	Characterize the epidemiology of the HPP population.	To characterize the epidemiology of the HPP population. Inclusion of all classifications of HPP is planned: pediatric-onset (perinatal-, infantile-, and juvenile-onset), adult-onset, benign perinatal, and odontohypophosphatasia.	1 year
Primary	Long-Term Safety and Effectiveness of Asfotase Alfa	To collect and evaluate long-term safety and effectiveness data in HPP patients who have/are receiving treatment with asfotase alfa.	1 year