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

NCT04195763 | Completed FDA Drug

• 1 other identifier: ALX-HPP-503
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

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™.

Conditions

Hypophosphatasia

Keywords

Pediatric-onset hypophosphatasia; Pediatric-onset; Hypophosphatasia; HPP; Patient reported outcome

Outcome Measures

Primary Outcomes (1)

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

  Baseline, up to 12 months

Study Arms (1)

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

Interventions

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.

Also known as: Strensiq®

Participants with Pediatric-onset HPP

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• ≥ 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead
• Xcenda, LLC: collaborator

Study Sites (1)

Xcenda, LLC

Palm Harbor, Florida, 34685, United States

Location

MeSH Terms

Conditions

Hypophosphatasia

Interventions

asfotase alfa

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 10, 2019
• First Posted: December 12, 2019
• Study Start: November 6, 2019
• Primary Completion: April 12, 2024
• Study Completion: April 12, 2024
• Last Updated: July 10, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)