Clinical trial NCT05234567: A Prospective Sub-Study of the Global Hypophosphatasia Registry
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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hypophosphatasia
Keywords: Pediatric Onset
HPP
Asfotase alfa
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2022-02-10
Enrollment: 30
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with HPP

Brief 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 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.

Primary outcomes:
measure: Occurrence Of Immune-mediated Loss Of Effectiveness According To The Treating Physician
timeFrame: Up to 5 years
measure: Occurrence Of Immune-mediated Serious Adverse Events
timeFrame: Up to 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Hartford
country: United States
facility: Clinical Trial Site
state: Connecticut
city: Chicago
country: United States
facility: Clinical Trial Site
state: Illinois
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Kansas City
country: United States
facility: Clinical Trial Site
state: Missouri
city: Mineola
country: United States
facility: Clinical Trial Site
state: New York
city: Cincinnati
country: United States
facility: Clinical Trial Site
state: Ohio
city: Columbus
country: United States
facility: Clinical Trial Site
state: Ohio
city: Pittsburgh
country: United States
facility: Clinical Trial Site
state: Pennsylvania
city: Nashville
country: United States
facility: Clinical Trial Site
state: Tennessee
city: Salt Lake City
country: United States
facility: Clinical Trial Site
state: Utah
city: Charlottesville
country: United States
facility: Clinical Trial Site
state: Virginia
city: Madison
country: United States
facility: Clinical Trial Site
state: Wisconsin
Study officials:
References: