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

March 15, 2024 updated by: Alexion Pharmaceuticals, Inc.

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

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™.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Estimated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Palm Harbor, Florida, United States, 34685
        • Xcenda, LLC

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 99 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Male or female participants ≥18 years of age who have been diagnosed with pediatric-onset HPP, have been newly prescribed treatment with asfotase alfa, and are registered in the patient support program managed by OneSource will be invited to participate.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Participants with Pediatric-onset HPP
Adult participants diagnosed with pediatric-onset HPP, newly prescribed treatment with asfotase alfa, and registered in the patient support program managed by OneSource.
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.
Other Names:
  • Strensiq®

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals, Inc.

Collaborators

Xcenda, LLC

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 6, 2019

Primary Completion (Estimated)

May 3, 2024

Study Completion (Estimated)

May 3, 2024

Study Registration Dates

First Submitted

December 10, 2019

First Submitted That Met QC Criteria

December 10, 2019

First Posted (Actual)

December 12, 2019

Study Record Updates

Last Update Posted (Actual)

March 18, 2024

Last Update Submitted That Met QC Criteria

March 15, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALX-HPP-503

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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