Metabolic DiseasesOBSERVANCE MHMNCT07356557
Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital
An observational study of Metabolic Diseases, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 7 to 20 years
- Conditions the study is for
- Metabolic Diseases
- What is being tested?
- Questionnaire and Calculation of the metabolic balance score
- Type of treatment
- Pill or oral medicine
- How many people
- 200 participants
- Where
- 1 siteFrance
- Study dates
- 23 Feb 2026to 23 Feb 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Hôpital Necker-Enfants Malades
Paris, 75015
Margaux GASCHIGNARD, M.D., 01 44 49 40 23 ext. +33, margaux.gaschignard@aphp.fr
Pascale M.D., PhD de Lonlay, 01 44 49 40 23 ext. +33, pascale.delonlay@aphp.fr
About this study
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.
Read the 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.
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
- Being tested
- Questionnaire, Calculation of the metabolic balance score
- Keywords
- Metabolic disease, Daily oral drug treatment, Medication adherence, Therapeutic education workshop
- Medical subject headings
- Metabolic Diseases, Medication Adherence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QuestionnaireOther
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 scoreOther
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Other: Questionnaire; Other: Calculation of the metabolic balance score |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of therapeutic adherence
Therapeutic adherence score, established from the declarations of patients and their parents to the study questionnaire.
Measured: Time 0
Secondary outcomes (9)
Number of treatments taken each day
Quantification of the number of medications taken daily based on the declarations of patients and their parents to the study questionnaire.
Measured: Time 0
Knowledge of the disease
Assessment of patients' and their parents' knowledge of the disease establish from the declarations of patients and their parents to the study questionnaire.
Measured: Time 0
Knowledge of treatments
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.
Measured: Time 0
Correlation between the therapeutic adherence score and the number of medications taken daily by patients
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.
Measured: Time 0
Correlation between the therapeutic adherence score and the age of patients
Therapeutic adherence score, established from the declarations of patients and their parents to the study questionnaire.
Measured: Time 0
Correlation between the therapeutic adherence score and patients' and their parents' knowledge of the disease
The therapeutic adherence score and the knowledge of the disease are established from the declarations of patients and their parents to the study questionnaire.
Measured: Time 0
Correlation between the therapeutic adherence score and patients' and their parents' knowledge of treatments
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.
Measured: Time 0
Correlation between therapeutic adherence score and metabolic balance for patients cared for urea cycle deficiency
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.
Measured: Time 0
Assessment of medication adherence in the event of a travel
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.
Measured: Time 0
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2026
- Study start23 Feb 2026
- Primary completion23 Feb 2028 (estimated)
- Study completion23 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Metabolic DiseasesStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- Margaux MD GASCHIGNARD, M.D., Principal investigator, Assistance Publique - Hôpitaux de ParisPascale de LONLAY, M.D., PhD, Study director, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07356557
- Sponsor's protocol ID
- APHP251413
- Acronym
- OBSERVANCE MHM
- Status last verified
- Jun 2026
- First submitted
- 17 Dec 2025
- Last updated
- 30 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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.
Study at a glance
From the registry record, last updated by the sponsor on 30 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital
- ClinicalTrials.gov ID
- NCT07356557
- Acronym / sponsor ID
- OBSERVANCE MHM / APHP251413
- Conditions
- Metabolic Diseases
- Keywords
- Metabolic disease, Daily oral drug treatment, Medication adherence, Therapeutic education workshop
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Study start
- 23 Feb 2026 (actual)
- Primary completion
- 23 Feb 2028 (estimated)
- Study completion
- 23 Feb 2028 (estimated)
- First posted
- 21 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients | – | Other: Questionnaire, Other: Calculation of the metabolic balance scorePatients, 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. |
Who may qualify
- Ages
- 7 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| QuestionnaireA 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 | Patients |
| Calculation of the metabolic balance scoreFor 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. | Other | Patients |
Primary outcome
Assessment of therapeutic adherence
Time frame: Time 0
Therapeutic adherence score, established from the declarations of patients and their parents to the study questionnaire.
9 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jun 2026.
- BackgroundModi AC, et al. "Adolescent adherence to antiepileptic drug therapy: determinants and clinical outcomes." JAMA, 2011.
- BackgroundMaestri NE, et al. "Outcome of urea cycle disorder patients: importance of metabolic control and compliance." Journal of Pediatrics, 1996.
- BackgroundTen Hoedt AE, et al. "Adherence to treatment in phenylketonuria: challenges and improvements." Molecular Genetics and Metabolism, 2011.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Margaux MD GASCHIGNARD, M.D. principal investigator
Assistance Publique - Hôpitaux de Paris
Pascale de LONLAY, M.D., PhD study director
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Metabolic Diseases, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 30 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Margaux GASCHIGNARD, M.D. (01 44 49 40 23 ext. +33, margaux.gaschignard@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessment of therapeutic adherence (time frame: Time 0). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07356557, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jun 2026.