Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital
Assistance Publique - Hôpitaux de Paris
200 participants
Feb 23, 2026
OBSERVATIONAL
Conditions
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.
Eligibility
Inclusion Criteria3
- All patients followed in the Hereditary Metabolic Diseases department of Necker Hospital during their visit to the department for their usual care and having a specific daily oral medication treatment.
- Children aged at least 7 years and adolescents/young adults
- Holders of parental authority and children or adolescents or adults' patients informed and consenting to participate in the study
Exclusion Criteria3
- Metabolic disease without oral medication (intravenous treatments, amino acid mixtures, and dietary regimens are not evaluated).
- Patient and parents not proficient in French.
- Refusal by the patient's holders of parental authority or adult patient to participate in the study and/or refusal of the child/adolescent.
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
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.
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.
Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07356557