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Completed

NCT Number: NCT04195763

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

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™.

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Key information

Conditions

Hypophosphatasia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year–99 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Xcenda, LLC

Palm Harbor, Florida, 34685, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

asfotase alfa

Drug

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.

Other names: Strensiq®

Primary outcomes

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

    Time frame: Baseline, up to 12 months

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Collaborators

  • Xcenda, LLC

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2024
Study completion
2024
First posted
Dec 12, 2019
Registry last updated
Jul 10, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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