A Prospective Sub-Study of the Global Hypophosphatasia Registry

• Register: ClinicalTrials.gov
• Last refreshed on: 10 March 2025
• Main ID: NCT05234567
• Date of registration: 01/02/2022
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals, Inc.
• Public title: A Prospective Sub-Study of the Global Hypophosphatasia Registry
• Scientific 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
• Date of first enrolment: August 25, 2022
• Target sample size: 30
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT05234567
• Study type: Observational

Countries of recruitment

United States

Contacts

• Name: Alexion Pharmaceuticals, Inc. (Sponsor)

Key inclusion & exclusion criteria

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.


Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Hypophosphatasia

Intervention(s)

Biological: Asfotase Alfa

Primary Outcome(s)

Occurrence Of Immune-mediated Serious Adverse Events [Time Frame: Up to 5 years]

Occurrence Of Immune-mediated Loss Of Effectiveness According To The Treating Physician [Time Frame: Up to 5 years]

Secondary ID(s)

ALX-HPP-501s

Source(s) of Monetary Support

Please refer to primary and secondary sponsors