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Orphan Drugs for Inherited Metabolic Diseases

NCT05818566 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 39

Last updated 2024-12-04

No results posted yet for this study

Summary

The aim of this study is to report and describe all the patients with confirmed diagnosis of inherited metabolic disease (IMD) treated with orphan medicinal products (OMPs) in a cohort of adult patients followed in a reference center for rare diseases (Lausanne University Hospital, CHUV) from 2017-2022.

Conditions

  • Inborn Errors of Metabolism

Sponsors & Collaborators

  • University of Lausanne

    lead OTHER

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-10-01
Primary Completion
2024-05-01
Completion
2024-10-01

Countries

  • Switzerland

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 NCT05818566 on ClinicalTrials.gov