NCT05234567 A Prospective Sub-Study of the Global Hypophosphatasia Registry
| NCT ID | NCT05234567 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Alexion Pharmaceuticals, Inc. |
| Condition | Hypophosphatasia |
| Study Type | OBSERVATIONAL |
| Enrollment | 30 participants |
| Start Date | 2022-08-25 |
| Primary Completion | 2028-07-18 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 30 participants in total. It began in 2022-08-25 with a primary completion date of 2028-07-18.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05234567 clinical trial?
This trial is open to participants of all sexes, studying Hypophosphatasia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05234567 currently recruiting?
Yes, NCT05234567 is actively recruiting participants. Contact the research team at clinicaltrials@alexion.com for enrollment information.
Where is the NCT05234567 trial being conducted?
This trial is being conducted at Hartford, United States, Chicago, United States, Boston, United States, Kansas City, United States and 8 additional locations.
Who is sponsoring the NCT05234567 clinical trial?
NCT05234567 is sponsored by Alexion Pharmaceuticals, Inc.. The trial plans to enroll 30 participants.