CompletedNot Applicable

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

United States50 participantsStarted 2019-11-06

Plain-language 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™.

Who can participate

Age range18 Years – 99 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

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

Timeframe: Baseline, up to 12 months

Trial details

NCT IDNCT04195763

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2024-04-12

View on ClinicalTrials.gov More Hypophosphatasia trials