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A Prospective Sub-Study of the Global Hypophosphatasia Registry | Clinical Trials | Clareo Health

RECRUITING

A Prospective Sub-Study of the Global Hypophosphatasia Registry

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

NCT05234567•Alexion Pharmaceuticals, Inc.

View on ClinicalTrials.gov

Summary

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.

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

Inclusion 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.

Exclusion Criteria

•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.

Locations (12)

Clinical Trial Site

Hartford, Connecticut, United States

Clinical Trial Site

Chicago, Illinois, United States

Clinical Trial Site

Boston, Massachusetts, United States

Clinical Trial Site

Kansas City, Missouri, United States

Clinical Trial Site

Mineola, New York, United States

Clinical Trial Site

Cincinnati, Ohio, United States

Clinical Trial Site

Columbus, Ohio, United States

Clinical Trial Site

Pittsburgh, Pennsylvania, United States

Clinical Trial Site

Nashville, Tennessee, United States

Clinical Trial Site

Salt Lake City, Utah, United States

Key Dates

Start Date

August 25, 2022

Primary Completion Date

July 18, 2028

Completion Date

July 18, 2028

Last Updated

February 9, 2026

Enrollment

ESTIMATED participants

Conditions

Hypophosphatasia

Interventions

Asfotase Alfa

BIOLOGICAL

Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356 clinicaltrials@alexion.com

The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in Russia

NCT07390240

Hypophosphatasia

View study

RECRUITINGPHASE1/PHASE2

A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1

NCT07179640

Hypophosphatasia (HPP)

View study

ACTIVE_NOT_RECRUITINGPHASE3

Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 9, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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A Prospective Sub-Study of the Global Hypophosphatasia Registry

Inclusion Criteria

Exclusion Criteria

The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in Russia

A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1

Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa

Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia

Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)

Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)