RECRUITING

A Prospective Sub-Study of the Global Hypophosphatasia Registry

Conditions

Hypophosphatasia Pediatric Onset HPP Asfotase alfa

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2022-08-25

Study Completion:2029-08-19

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05234567

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.

Ages Eligible for Study:Not specified

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.

Inclusion Criteria

Exclusion 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.

Contacts and Locations

Study Contact

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356

clinicaltrials@alexion.com

Study Locations (Sites)

Clinical Trial Site

Hartford, Connecticut, 06106

United States

Clinical Trial Site

Chicago, Illinois, 60611

United States

Clinical Trial Site

Boston, Massachusetts, 02122

United States

Clinical Trial Site

Kansas City, Missouri, 64108

United States

Clinical Trial Site

Mineola, New York, 11501

United States

Clinical Trial Site

Cincinnati, Ohio, 45229

United States

Clinical Trial Site

Columbus, Ohio, 43203

United States

Clinical Trial Site

Pittsburgh, Pennsylvania, 15224

United States

Clinical Trial Site

Nashville, Tennessee, 37112

United States

Clinical Trial Site

Salt Lake City, Utah, 84108

United States

Clinical Trial Site

Charlottesville, Virginia, 22903

United States

Clinical Trial Site

Madison, Wisconsin, 53792

United States

Collaborators and Investigators

Sponsor: Alexion Pharmaceuticals, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-08-25

Study Completion Date2029-08-19

Study Record Updates

Study Start Date2022-08-25

Study Completion Date2029-08-19

Terms related to this study

Keywords Provided by Researchers

Pediatric Onset
HPP
Asfotase alfa

Additional Relevant MeSH Terms

Hypophosphatasia