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

A Prospective Study to Evaluate the Patient Reported Quality of Life Prior to and After Strensiq® Treatment in Adults With Pediatric Onset Hypophosphatasia

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04195763

Enrollment

Registered

2019-12-12

Start date

2019-11-06

Completion date

2024-04-12

Last updated

2024-07-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hypophosphatasia

Keywords

Pediatric-onset hypophosphatasia, Pediatric-onset, Hypophosphatasia, HPP, Patient reported outcome

Brief summary

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™.

Detailed description

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.

Interventions

DRUGasfotase alfa

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.

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to 99 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Change From Baseline In Patient Reported Outcomes (PROs) Questionnaire Scores	Baseline, up to 12 months

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026