Trials / Completed
CompletedNCT03418389
Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia
An Observational, Longitudinal Study to Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Pediatric-Onset Hypophosphatasia
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 23 (actual)
- Sponsor
- Dr. Lothar Seefried · Academic / Other
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
Hypophosphatasia is a rare inherited metabolic disorder due to inactivating mutations of the ALPL-Gene. Particularly among adult patients, clinical manifestation exhibits a broad range of signs and symptoms, most commonly associated with musculoskeletal disabilities and compromised quality of life. Enzyme replacement therapy with Asfotase alfa (AA) is available and approved for patients with pediatric onset of the disease. This single-center observational cohort study aims at collecting clinical routine data regarding the course treatment, quality of life and physical performance in patients treated with Asfotase alfa in line with the label for pediatric-onset hypophosphatasia.
Conditions
Timeline
- Start date
- 2018-09-05
- Primary completion
- 2021-05-31
- Completion
- 2021-05-31
- First posted
- 2018-02-01
- Last updated
- 2022-07-12
Locations
1 site across 1 country: Germany
Source: ClinicalTrials.gov record NCT03418389. Inclusion in this directory is not an endorsement.