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

Study Identifier:
ALX-HPP-503
ClinicalTrials.gov Identifier:
NCT04195763
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: asfotase alfa
Date
Nov 2019 - Apr 2024
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 99 Years
Requirements Information
Inclusion and Exclusion Criteria
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
Sex
Female & Male
Age
18 - 99 Years

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: asfotase alfa
Date
Nov 2019 - Apr 2024
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 99 Years years
Requirements Information
Inclusion and Exclusion Criteria
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

Protocol 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™.

Trial Locations

Location
Status
Location
Xcenda, LLC
Palm Harbor, Florida, United States, 34685
Status
N/A