NCT04195763

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
• Phase: —
• Study type: Observational
• Enrollment: 50 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 99 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hypophosphatasia

Interventions

Type	Name	Description
DRUG	asfotase 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.

Timeline

• Start date: 2019-11-06
• Primary completion: 2024-04-12
• Completion: 2024-04-12
• First posted: 2019-12-12
• Last updated: 2024-07-10

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study