Hypophosphatasia
Conditions
Keywords
Pediatric Onset, HPP, Asfotase alfa
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.
Interventions
BIOLOGICALAsfotase Alfa
All participants will receive asfotase alfa subcutaneously per standard of care. Unless otherwise specified per the Physician's standard of care, participants aged \< 2 years are recommended for a clinic visit approximately every 3 months after Enrollment until 2 years of age, after which they should have a clinic visit approximately every 6 months. Participants should be followed for 5 years, as possible.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Occurrence Of Immune-mediated Loss Of Effectiveness According To The Treating Physician | Up to 5 years | This will be based on clinical and biochemical assessments as well as positive anti-drug antibodies and positive neutralizing antibodies. |
| Occurrence Of Immune-mediated Serious Adverse Events | Up to 5 years | These serious adverse events will include serious hypersensitivity reactions and anaphylaxis. |
Countries
United States
Contacts
CONTACTAlexion Pharmaceuticals, Inc. (Sponsor)
Outcome results
None listed