We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04195763
Recruitment Status : Unknown
Verified December 2019 by Alexion Pharmaceuticals.
Recruitment status was:  Enrolling by invitation
First Posted : December 12, 2019
Last Update Posted : December 12, 2019
Sponsor:
Collaborator:
Xcenda, LLC
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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™.

Condition or disease Intervention/treatment
Hypophosphatasia Drug: asfotase alfa

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.

Layout table for study information
Study Type : Observational
Estimated Enrollment : 100 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Prospective Study to Evaluate the Patient Reported Quality of Life Prior to and After Strensiq® Treatment in Adults With Pediatric Onset Hypophosphatasia
Actual Study Start Date : November 6, 2019
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2021

Resource links provided by the National Library of Medicine


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 Name: Strensiq®




Primary Outcome Measures :
  1. Change From Baseline In Patient Reported Outcomes (PROs) Questionnaire Scores [ Time Frame: Baseline, up to 12 months ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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.
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04195763


Locations
Layout table for location information
United States, Florida
Xcenda, LLC
Palm Harbor, Florida, United States, 34685
Sponsors and Collaborators
Alexion Pharmaceuticals
Xcenda, LLC
Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04195763    
Other Study ID Numbers: ALX-HPP-503
First Posted: December 12, 2019    Key Record Dates
Last Update Posted: December 12, 2019
Last Verified: December 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
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
Keywords provided by Alexion Pharmaceuticals:
Pediatric-onset hypophosphatasia
Pediatric-onset
Hypophosphatasia
HPP
Patient reported outcome
Additional relevant MeSH terms:
Layout table for MeSH terms
Hypophosphatasia
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases