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

Alexion Pharmaceuticals

Status: Completed

Conditions: Hypophosphatasia

Treatments: Drug: asfotase alfa

Study type: Observational

Funder types: Industry; Other

Identifiers

NCT04195763

ALX-HPP-503

Details and patient eligibility

About

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™.

Full 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.

Enrollment: 50 patients

Sex: All

Ages: 18 to 99 years old

Volunteers: No Healthy Volunteers

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

Trial design

50 participants in 1 patient group

Participants with Pediatric-onset HPP

Description:

Adult participants diagnosed with pediatric-onset HPP, newly prescribed treatment with asfotase alfa, and registered in the patient support program managed by OneSource.

Treatment:

Drug: asfotase alfa