A Prospective Sub-Study of the Global Hypophosphatasia Registry
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.
Conditions:
🦠 Hypophosphatasia
πŸ—“οΈ Study Start (Actual) 17 March 2022
πŸ—“οΈ Primary Completion (Estimated) 30 June 2028
βœ… Study Completion (Estimated) 30 June 2028
πŸ‘₯ Enrollment (Estimated) 40
πŸ”¬ Study Type OBSERVATIONAL
πŸ“Š Phase N/A
Locations:
πŸ“ Hartford, Connecticut, United States
πŸ“ Chicago, Illinois, United States
πŸ“ Boston, Massachusetts, United States
πŸ“ Kansas City, Missouri, United States
πŸ“ Las Vegas, Nevada, United States
πŸ“ Mineola, New York, United States
πŸ“ Cincinnati, Ohio, United States
πŸ“ Columbus, Ohio, United States
πŸ“ Philadelphia, Pennsylvania, United States
πŸ“ Pittsburgh, Pennsylvania, United States
πŸ“ Nashville, Tennessee, United States
πŸ“ Salt Lake City, Utah, United States
πŸ“ Charlottesville, Virginia, United States

πŸ“‹ Eligibility Criteria

Description

    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-naΓ―ve) or the Physician has decided to resume (not treatment-naΓ―ve) or start (treatment-naΓ―ve) 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.
Ages Eligible for Study: N/A to N/A (CHILD, ADULT, OLDER_ADULT)
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No

πŸ—“οΈ Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

  • First Submitted 1 February 2022
  • First Submitted that Met QC Criteria 1 February 2022
  • First Posted 10 February 2022

Study Record Updates

  • Last Update Submitted that Met QC Criteria 11 July 2023
  • Last Update Posted 12 July 2023
  • Last Verified July 2023