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

Completed

• Conditions: Hypophosphatasia
• Interventions: Drug: asfotase alfa

• Registration Number: NCT04195763
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

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.

Study Timeline

• Study Start: 2019-11-06
• Study First Submitted: 2019-12-10
• Study First Submitted QC: 2019-12-10
• Study First Posted: 2019-12-12
• Primary Completion: 2024-04-12
• Study Completion: 2024-04-12
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-09
• Last Update Posted: 2024-07-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 50

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Participants with Pediatric-onset HPP	asfotase alfa	Adult participants diagnosed with pediatric-onset HPP, newly prescribed treatment with asfotase alfa, and registered in the patient support program managed by OneSource.

Primary Outcome Measures

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

Trial Locations

Locations (1)

Xcenda, LLC; 🇺🇸 Palm Harbor, Florida, United States