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NCT Number: NCT07356557

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

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.

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Key information

Conditions

Metabolic Diseases Behavior Health Behavior Medication Adherence Nutritional and Metabolic Diseases Patient Acceptance of Health Care Patient Compliance Treatment Adherence and Compliance

Age range

7 year–20 year

Sex eligibility

All sexes

Study type

Observational

Primary location

About this study

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.

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Questionnaire

Other

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.

Calculation of the metabolic balance score

Other

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 outcomes

  1. Assessment of therapeutic adherence

    Time frame: Time 0

    Therapeutic adherence score, established from the declarations of patients and their parents to the study questionnaire.

Secondary outcomes

  1. Number of treatments taken each day

    Time frame: Time 0

    Quantification of the number of medications taken daily based on the declarations of patients and their parents to the study questionnaire.

  2. Knowledge of the disease

    Time frame: Time 0

    Assessment of patients' and their parents' knowledge of the disease establish from the declarations of patients and their parents to the study questionnaire.

  3. Knowledge of treatments

    Time frame: Time 0

    Accordance of patients' and their parents' knowledge of treatments, based on the declarations of patients and their parents in the study questionnaire and the patient's prescriptions in the medical record.

  4. Correlation between the therapeutic adherence score and the number of medications taken daily by patients

    Time frame: Time 0

    The therapeutic adherence score and the number of medications taken daily are established from the declarations of patients and their parents to the study questionnaire.

  5. Correlation between the therapeutic adherence score and the age of patients

    Time frame: Time 0

    Therapeutic adherence score, established from the declarations of patients and their parents to the study questionnaire.

  6. Correlation between the therapeutic adherence score and patients' and their parents' knowledge of the disease

    Time frame: Time 0

    The therapeutic adherence score and the knowledge of the disease are established from the declarations of patients and their parents to the study questionnaire.

  7. Correlation between the therapeutic adherence score and patients' and their parents' knowledge of treatments

    Time frame: Time 0

    The therapeutic adherence score and the knowledge of treatments are established from the declarations of patients and their parents to the study questionnaire and on the patient's prescription.

  8. Correlation between therapeutic adherence score and metabolic balance for patients cared for urea cycle deficiency

    Time frame: Time 0

    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 based on the following clinical data:

    • Number of ammonia levels > 100 µmol/L,
    • Mean and median follow-up ammonia levels,
    • Number of glutamine levels > 1000 µmol/L,
    • Number of hospitalizations for decompensation.

  9. Assessment of medication adherence in the event of a travel

    Time frame: Time 0

    Assessment of patients' and their parents' medication adherence in the event of a travel, established from the declarations of patients and their parents to the study questionnaire.

Study contacts

Contact information is provided by the study sponsor or research team.

Hélène Morel

CONTACT

[email protected]

01 71 19 63 46 ext. +33

Margaux GASCHIGNARD, M.D.

CONTACT

[email protected]

01 44 49 40 23 ext. +33

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Collaborators

  • URC-CIC Paris Descartes Necker Cochin

Registry information

Acronym: OBSERVANCE MHM

Important dates

Study start
2026
Primary completion
2028
Study completion
2028
First posted
Jan 21, 2026
Registry last updated
Jun 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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