A Prospective Sub-Study of the Global Hypophosphatasia Registry

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

First listed

Feb 10, 2022

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

• Alexion Pharmaceuticals, Inc. (Sponsor)
  1-855-752-2356
  [email protected]

Locations (12)

Find similar trials in Hartford, CT

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