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Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital

NCT07356557 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 200

Last updated 2026-03-10

No results posted yet for this study

Summary

The purpose of this study is to evaluate treatment adherence among patients followed in the Department of Inherited Metabolic Diseases at Necker Hospital, in order to assess the need for implementing a therapeutic education workshop focused on medication adherence.

Conditions

Interventions

OTHER

Questionnaire

A pseudonymized questionnaire assessing treatment adherence, understanding of the illness, knowledge of medications, and the number of oral medications taken is given to patients and/or their parents during a follow-up visit, whether in a consultation, day hospital, or conventional hospitalization. It is completed by parents and children aged 7 and older, as well as by adolescents and young adults. The responses will not be disclosed to the physicians in the department.

OTHER

Calculation of the metabolic balance score

For patients with urea cycle deficiency, a metabolic balance score will be calculated independently from retrospective data from the medical record, in the last two years preceding the completion of the questionnaire.

Sponsors & Collaborators

  • URC-CIC Paris Descartes Necker Cochin

    collaborator OTHER

  • Assistance Publique - Hôpitaux de Paris

    lead OTHER

Principal Investigators

  • Margaux MD GASCHIGNARD, M.D. · Assistance Publique - Hôpitaux de Paris

  • Pascale de LONLAY, M.D., PhD · Assistance Publique - Hôpitaux de Paris

Eligibility

Min Age
7 Years
Max Age
20 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-02-23
Primary Completion
2028-02-23
Completion
2028-02-23

Countries

  • France

Study Locations

More Related Trials

Entities

Diseases

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 NCT07356557 on ClinicalTrials.gov