A Prospective Sub-Study of the Global Hypophosphatasia Registry

Description

In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and potentially 1 or 2 other countries.

Conditions

Hypophosphatasia

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Study Overview

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Study Details

Study overview

In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and potentially 1 or 2 other countries.

A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants With Hypophosphatasia

A Prospective Sub-Study of the Global Hypophosphatasia Registry

Condition
Hypophosphatasia
Intervention / Treatment

-

Contacts and Locations

Hartford

Clinical Trial Site, Hartford, Connecticut, United States, 06106

Chicago

Clinical Trial Site, Chicago, Illinois, United States, 60611

Boston

Clinical Trial Site, Boston, Massachusetts, United States, 02122

Kansas City

Clinical Trial Site, Kansas City, Missouri, United States, 64108

Mineola

Clinical Trial Site, Mineola, New York, United States, 11501

Cincinnati

Clinical Trial Site, Cincinnati, Ohio, United States, 45229

Columbus

Clinical Trial Site, Columbus, Ohio, United States, 43203

Pittsburgh

Clinical Trial Site, Pittsburgh, Pennsylvania, United States, 15224

Nashville

Clinical Trial Site, Nashville, Tennessee, United States, 37112

Salt Lake City

Clinical Trial Site, Salt Lake City, Utah, United States, 84108

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Any age or sex with a confirmed diagnosis of pediatric-onset HPP (that is, first HPP sign or symptom presented at \< 18 years of age).
  • * Currently receiving asfotase alfa treatment at Enrollment (not treatment-naïve) or the Physician has decided to resume (not treatment-naïve) or start (treatment-naïve) the participant's asfotase alfa treatment within 6 months after Enrollment.
  • * Participant must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, and a documented ALPL gene mutation (Note: An exception is made for infants with clinical features of HPP plus low ALP who need to start asfotase alfa treatment right away, at the Physician's discretion, but do not yet have a genetic result. In this case, ALPL gene documentation is not required at the time of sub-study enrollment but should be documented within 6 months after Enrollment).
  • * Participant or participant's parent/legally authorized representative is able to read and/or understand the informed consent and study questionnaires in the local language.
  • * Participant or participant's parent/legally authorized representative must be willing and able to give signed informed consent for this sub-study, and the participant must be willing to give written informed assent, if appropriate and required by local regulations.
  • * Currently participating in an Alexion-sponsored interventional clinical study. Participants who have concluded participation in an Alexion-sponsored asfotase alfa clinical study are eligible to enroll in this sub-study.

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Alexion Pharmaceuticals, Inc.,

Study Record Dates

2029-08-19