Registry of Patients With Hypophosphatasia

Alexion Pharmaceuticals

Status: Invitation-only

Conditions: Hypophosphatasia (HPP)

Study type: Observational

Funder types: Industry

Identifiers

NCT02306720

ALX-HPP-501

Details and patient eligibility

About

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.

Full 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

Enrollment: 900 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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.