Ignite Creation Date:
2026-03-26 @ 3:16 PM
Ignite Modification Date:
2026-03-30 @ 2:26 AM
Study NCT ID:
NCT07356557
Status:
RECRUITING
Last Update Posted:
2026-03-10
First Post:
2025-12-17
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'conditionBrowseModule': {'meshes': [{'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D055118', 'term': 'Medication Adherence'}], 'ancestors': [{'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D010349', 'term': 'Patient Compliance'}, {'id': 'D010342', 'term': 'Patient Acceptance of Health Care'}, {'id': 'D000074822', 'term': 'Treatment Adherence and Compliance'}, {'id': 'D015438', 'term': 'Health Behavior'}, {'id': 'D001519', 'term': 'Behavior'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D011795', 'term': 'Surveys and Questionnaires'}], 'ancestors': [{'id': 'D003625', 'term': 'Data Collection'}, {'id': 'D004812', 'term': 'Epidemiologic Methods'}, {'id': 'D008919', 'term': 'Investigative Techniques'}, {'id': 'D017531', 'term': 'Health Care Evaluation Mechanisms'}, {'id': 'D011787', 'term': 'Quality of Health Care'}, {'id': 'D017530', 'term': 'Health Care Quality, Access, and Evaluation'}, {'id': 'D011634', 'term': 'Public Health'}, {'id': 'D004778', 'term': 'Environment and Public Health'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 200}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2026-02-23', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2028-02-23', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-03-09', 'studyFirstSubmitDate': '2025-12-17', 'studyFirstSubmitQcDate': '2026-01-14', 'lastUpdatePostDateStruct': {'date': '2026-03-10', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-01-21', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-02-23', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Assessment of therapeutic adherence', 'timeFrame': 'Time 0', 'description': 'Therapeutic adherence score, established from the declarations of patients and their parents to the study questionnaire.'}], 'secondaryOutcomes': [{'measure': 'Number of treatments taken each day', 'timeFrame': 'Time 0', 'description': 'Quantification of the number of medications taken daily based on the declarations of patients and their parents to the study questionnaire.'}, {'measure': 'Knowledge of the disease', 'timeFrame': 'Time 0', 'description': "Assessment of patients' and their parents' knowledge of the disease establish from the declarations of patients and their parents to the study questionnaire."}, {'measure': 'Knowledge of treatments', 'timeFrame': 'Time 0', 'description': "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."}, {'measure': 'Correlation between the therapeutic adherence score and the number of medications taken daily by patients', 'timeFrame': 'Time 0', 'description': '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.'}, {'measure': 'Correlation between the therapeutic adherence score and the age of patients', 'timeFrame': 'Time 0', 'description': 'Therapeutic adherence score, established from the declarations of patients and their parents to the study questionnaire.'}, {'measure': "Correlation between the therapeutic adherence score and patients' and their parents' knowledge of the disease", 'timeFrame': 'Time 0', 'description': 'The therapeutic adherence score and the knowledge of the disease are established from the declarations of patients and their parents to the study questionnaire.'}, {'measure': "Correlation between the therapeutic adherence score and patients' and their parents' knowledge of treatments", 'timeFrame': 'Time 0', 'description': "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."}, {'measure': 'Correlation between therapeutic adherence score and metabolic balance for patients cared for urea cycle deficiency', 'timeFrame': 'Time 0', 'description': '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:\n\n* Number of ammonia levels \\> 100 µmol/L,\n* Mean and median follow-up ammonia levels,\n* Number of glutamine levels \\> 1000 µmol/L,\n* Number of hospitalizations for decompensation.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Metabolic disease', 'Daily oral drug treatment', 'Medication adherence', 'Therapeutic education workshop'], 'conditions': ['Metabolic Diseases']}, 'referencesModule': {'references': [{'type': 'BACKGROUND', 'citation': 'Modi AC, et al. "Adolescent adherence to antiepileptic drug therapy: determinants and clinical outcomes." JAMA, 2011.'}, {'type': 'BACKGROUND', 'citation': 'Maestri NE, et al. "Outcome of urea cycle disorder patients: importance of metabolic control and compliance." Journal of Pediatrics, 1996.'}, {'type': 'BACKGROUND', 'citation': 'Ten Hoedt AE, et al. "Adherence to treatment in phenylketonuria: challenges and improvements." Molecular Genetics and Metabolism, 2011.'}]}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.\n\nThe 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '20 Years', 'minimumAge': '7 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': '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.', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* 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.\n* Children aged at least 7 years and adolescents/young adults\n* Holders of parental authority and children or adolescents or adults' patients informed and consenting to participate in the study\n\nExclusion Criteria:\n\n* Metabolic disease without oral medication (intravenous treatments, amino acid mixtures, and dietary regimens are not evaluated).\n* Patient and parents not proficient in French.\n* Refusal by the patient's holders of parental authority or adult patient to participate in the study and/or refusal of the child/adolescent."}, 'identificationModule': {'nctId': 'NCT07356557', 'acronym': 'OBSERVANCE MHM', 'briefTitle': 'Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital', 'organization': {'class': 'OTHER', 'fullName': 'Assistance Publique - Hôpitaux de Paris'}, 'officialTitle': 'Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital', 'orgStudyIdInfo': {'id': 'APHP251413'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Patients', 'description': '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.', 'interventionNames': ['Other: Questionnaire', 'Other: Calculation of the metabolic balance score']}], 'interventions': [{'name': 'Questionnaire', 'type': 'OTHER', 'description': '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.', 'armGroupLabels': ['Patients']}, {'name': 'Calculation of the metabolic balance score', 'type': 'OTHER', 'description': '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.', 'armGroupLabels': ['Patients']}]}, 'contactsLocationsModule': {'locations': [{'zip': '75015', 'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Margaux GASCHIGNARD, M.D.', 'role': 'CONTACT', 'email': 'margaux.gaschignard@aphp.fr', 'phone': '01 44 49 40 23', 'phoneExt': '+33'}, {'name': 'Hélène Morel', 'role': 'CONTACT', 'email': 'helene.morel@aphp.fr', 'phone': '01 71 19 63 46', 'phoneExt': '+33'}], 'facility': 'Hôpital Necker-Enfants Malades', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}], 'centralContacts': [{'name': 'Margaux GASCHIGNARD, M.D.', 'role': 'CONTACT', 'email': 'margaux.gaschignard@aphp.fr', 'phone': '01 44 49 40 23', 'phoneExt': '+33'}, {'name': 'Hélène Morel', 'role': 'CONTACT', 'email': 'helene.morel@aphp.fr', 'phone': '01 71 19 63 46', 'phoneExt': '+33'}], 'overallOfficials': [{'name': 'Margaux MD GASCHIGNARD, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Assistance Publique - Hôpitaux de Paris'}, {'name': 'Pascale de LONLAY, M.D., PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Assistance Publique - Hôpitaux de Paris'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Assistance Publique - Hôpitaux de Paris', 'class': 'OTHER'}, 'collaborators': [{'name': 'URC-CIC Paris Descartes Necker Cochin', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}