Registry of Patients With Hypophosphatasia

N/AEnrolling By InvitationNCT02306720

Alexion Pharmaceuticals, Inc.1,571 enrolled

Overview

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.

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

Study Type

OBSERVATIONAL

Enrollment

1,571

Conditions

Hypophosphatasia (HPP)

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (65)

Outcomes

Primary Outcomes

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.

Time frame: 1 Year

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)

Time frame: 1 year

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.

Time frame: 1 year

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.

Time frame: 1 year

Data from ClinicalTrials.gov

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Research Site

Centennial, Colorado, United States

Research Site

Hartford, Connecticut, United States

Research Site

Tampa, Florida, United States

Research Site

Decatur, Georgia, United States

Research Site

Baltimore, Maryland, United States

Research Site

Boston, Massachusetts, United States

Research Site

Boston, Massachusetts, United States

Research Site

Minneapolis, Minnesota, United States

Research Site

Kansas City, Missouri, United States

Research Site

St Louis, Missouri, United States

...and 55 more locations