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Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia

NCT03418389 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 23

Last updated 2022-07-12

No results posted yet for this study

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

  • Hypophosphatasia

Sponsors & Collaborators

  • Alexion Pharmaceuticals, Inc.

    collaborator INDUSTRY

  • Dr. Lothar Seefried

    lead OTHER

Principal Investigators

  • Lothar Seefried, MD · Wuerzburg University, Orthopedic Institute, Clinical Trial Unit

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-09-05
Primary Completion
2021-05-31
Completion
2021-05-31

Countries

  • Germany

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03418389 on ClinicalTrials.gov