Recruiting NCT07356557

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

— Observational Metabolic Diseases

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied: The protocol lists: Questionnaire, Calculation of the metabolic balance score.
Who it may be relevant to: Registry conditions: Metabolic Diseases. Basic parameters: 7 years — 20 years · All.
What needs checking: Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place: France
Next step: Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Overview

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.

Detailed description

There is limited data in the literature regarding treatment adherence in children with metabolic disorders. A 2011 study on phenylketonuria highlighted a decline in dietary adherence during adolescence. Another study, dating from 1996 and focusing on urea cycle disorders, emphasized the importance of good treatment adherence for maintaining disease control. Furthermore, larger-scale research in epileptic patients has identified several barriers to treatment compliance, highlighting the crucial role of treatment adherence in improving disease control.

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.

Primary outcome measures

Assessment of therapeutic adherence [Time frame: Time 0]

Secondary outcome measures (9)

Number of treatments taken each day [Time frame: Time 0]
Knowledge of the disease [Time frame: Time 0]
Knowledge of treatments [Time frame: Time 0]
Correlation between the therapeutic adherence score and the number of medications taken daily by patients [Time frame: Time 0]
Correlation between the therapeutic adherence score and the age of patients [Time frame: Time 0]
Correlation between the therapeutic adherence score and patients' and their parents' knowledge of the disease [Time frame: Time 0]
Correlation between the therapeutic adherence score and patients' and their parents' knowledge of treatments [Time frame: Time 0]
Correlation between therapeutic adherence score and metabolic balance for patients cared for urea cycle deficiency [Time frame: Time 0]
Assessment of medication adherence in the event of a travel [Time frame: Time 0]

Eligibility criteria

Inclusion criteria

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 criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model: Case-only

Study locations

France · 1 center

Hôpital Necker-Enfants Malades — Paris

Publications

Modi AC, et al. "Adolescent adherence to antiepileptic drug therapy: determinants and clinical outcomes." JAMA, 2011.
Maestri NE, et al. "Outcome of urea cycle disorder patients: importance of metabolic control and compliance." Journal of Pediatrics, 1996.
Ten Hoedt AE, et al. "Adherence to treatment in phenylketonuria: challenges and improvements." Molecular Genetics and Metabolism, 2011.

Identifiers

NCT: NCT07356557 · APHP251413

Primary sources (government registries)

ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗