A Prospective Sub-Study of the Global Hypophosphatasia Registry

Part of paid clinical trials in Hartford, Connecticut.

Sponsor
Alexion Pharmaceuticals, Inc.
Study ID
NCT05234567
Status
Recruiting
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Conditions

  • Hypophosphatasia

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Asfotase Alfa — BIOLOGICAL
    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.

Study Details

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.

Key Dates

Start date
Aug 25, 2022
Status verified
Feb 2026
Primary completion
Jul 18, 2028
Completion
Jul 18, 2028

Study Design

Enrollment
30 participants (estimated)

Arms

  • Arm: Participants with Pediatric-onset HPP
    Each participant will be followed for a minimum of 5 years or, if applicable, until early withdrawal. Biochemical, clinical, imaging (if clinically indicated), and functional/quality of life outcomes relevant to HPP will be assessed.

Primary Outcome Measure

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

Central Contacts

Locations (12)

FacilityCityStateZIPSite coordinators
Clinical Trial SiteHartfordConnecticut06106-
Clinical Trial SiteChicagoIllinois60611-
Clinical Trial SiteBostonMassachusetts02122-
Clinical Trial SiteKansas CityMissouri64108-
Clinical Trial SiteMineolaNew York11501-
Clinical Trial SiteCincinnatiOhio45229-
Clinical Trial SiteColumbusOhio43203-
Clinical Trial SitePittsburghPennsylvania15224-
Clinical Trial SiteNashvilleTennessee37112-
Clinical Trial SiteSalt Lake CityUtah84108-
Clinical Trial SiteCharlottesvilleVirginia22903-
Clinical Trial SiteMadisonWisconsin53792-

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