Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)

N/ACompletedNCT04195763

Alexion Pharmaceuticals, Inc.50 enrolled

Overview

This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support program managed by OneSource™.

Participants consenting to participate in this study will be asked to complete study questionnaires by phone interviews upon consenting (Baseline), and up to 12 months following treatment initiation with asfotase alfa. Demographic and clinical characteristics of participants in the study will be characterized. This is an observational study and no intervention will be administered. Participants will be treated in accordance with standard of care.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Hypophosphatasia

Interventions

asfotase alfaDRUG

This is an observational study and no intervention will be administered. All participants will be treated by their physician in accordance with standard of care. All medications are commercially available and will be used as directed by the treating physician.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 99 Years

Medical Language ↔ Plain English

Inclusion Criteria: * ≥ 18 years old * Clinical diagnosis of pediatric-onset HPP * Naïve to asfotase alfa * Expected to begin treatment with asfotase alfa for HPP * Registered in OneSource * Willing and able to provide voluntary, verbal informed consent to participate in this study Exclusion Criteria: * Pregnant or breastfeeding * Unable to speak and understand English * Unable or unwilling to complete the study surveys via telephone interview at the protocol-required time points

Locations (1)

Xcenda, LLC

Palm Harbor, Florida, United States

Outcomes

Primary Outcomes

Change From Baseline In Patient Reported Outcomes (PROs) Questionnaire Scores

Time frame: Baseline, up to 12 months

Data from ClinicalTrials.gov

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