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

NCT07356557 | Recruiting

• 1 other identifier: APHP251413
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 1

Brief 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

Metabolic Diseases

Keywords

Metabolic disease; Daily oral drug treatment; Medication adherence; Therapeutic education workshop

Outcome Measures

Primary Outcomes (1)

• Assessment of therapeutic adherence

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

  Time 0

Secondary Outcomes (8)

• Number of treatments taken each day

  Time 0

• Knowledge of the disease

  Time 0

• Knowledge of treatments

  Time 0

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

  Time 0

• Correlation between the therapeutic adherence score and the age of patients

  Time 0

Study Arms (1)

Patients

Patients, children aged at least 7 years and adolescents/young adults followed in the pediatric department of hereditary metabolic diseases of Necker Hospital, having specific oral drug treatment.

Other: Questionnaire; Other: Calculation of the metabolic balance score

Interventions

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.

Patients

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.

Patients

Eligibility Criteria

• Age: 7 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Patients followed in the department of hereditary metabolic diseases at Necker Hospital, having specific oral drug treatment, and having a scheduled care appointment at Necker Hospital as part of their usual follow-up, during the study inclusion period.

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (1)

Hôpital Necker-Enfants Malades

Paris, 75015, France

RECRUITING

Related Publications (3)

• Modi AC, et al. "Adolescent adherence to antiepileptic drug therapy: determinants and clinical outcomes." JAMA, 2011.

  BACKGROUND

• Maestri NE, et al. "Outcome of urea cycle disorder patients: importance of metabolic control and compliance." Journal of Pediatrics, 1996.

  BACKGROUND

• Ten Hoedt AE, et al. "Adherence to treatment in phenylketonuria: challenges and improvements." Molecular Genetics and Metabolism, 2011.

  BACKGROUND

MeSH Terms

Conditions

Metabolic Diseases; Medication Adherence

Interventions

Surveys and Questionnaires

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Margaux MD GASCHIGNARD, M.D.

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

• Pascale de LONLAY, M.D., PhD

  Assistance Publique - Hôpitaux de Paris

  STUDY DIRECTOR

Central Study Contacts

Margaux GASCHIGNARD, M.D.

CONTACT

01 44 49 40 23; margaux.gaschignard@aphp.fr

Hélène Morel

CONTACT

01 71 19 63 46; helene.morel@aphp.fr

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 17, 2025
• First Posted: January 21, 2026
• Study Start: February 23, 2026
• Primary Completion (Estimated): February 23, 2028
• Study Completion (Estimated): February 23, 2028
• Last Updated: March 10, 2026

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)