Measuring how well patients follow daily oral medicines in Necker’s inherited metabolic diseases department
Official title: Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital
Run by Assistance Publique - Hôpitaux de Paris · NCT07356557 · Data updated 2026-11-12
This project will measure how well children, adolescents, and young adults with inherited metabolic diseases at Necker follow their daily oral medicines to see if a medication-education workshop is needed.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 7 Years to 20 Years |
| Sex | All |
| Enrollment | 200 (estimated) |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Locations | 1 site (Paris) |
| Status | Recruiting |
| Started | February 23, 2026 |
| Main results expected | February 23, 2028 |
| Study ends | February 23, 2028 |
| Registry record updated | June 30, 2026 |
| Trial ID | NCT07356557 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Patients aged 7 years and older who are followed at Necker for inherited metabolic diseases, take a daily oral medication, speak French, and consent to participate.
✕Probably not a fit
Patients without daily oral medications (those on only intravenous treatments, amino acid mixtures, or dietary regimens), those not proficient in French, or those who decline participation are not included and unlikely to benefit directly.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational project enrolls patients aged 7 years and older who are followed in Necker’s Department of Hereditary Metabolic Diseases and who take a daily oral medication. Participants complete a questionnaire and researchers calculate a metabolic balance score from clinical data and treatments to quantify adherence. The team will analyze adherence patterns across age groups and identify barriers linked to poorer compliance, with particular attention to adolescence. Results will determine whether implementing a targeted therapeutic education workshop on medication adherence is warranted.
Why it matters
Potential benefit: If successful, the project could lead to a targeted education workshop that improves medication adherence and helps maintain better metabolic control.
How similar studies have gone: Previous reports in phenylketonuria, urea cycle disorders, and epilepsy have documented adherence challenges—especially in adolescence—and suggest adherence-focused education can help, but large-scale data in inherited metabolic diseases are limited.
Who can join: the full rules
Show full inclusion / exclusion 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.
Where it's running
-
Hôpital Necker-Enfants Malades
Paris, France Recruiting
Study contacts
- Principal investigator: Margaux MD GASCHIGNARD, M.D. — Assistance Publique - Hôpitaux de Paris
- Study coordinator: Margaux GASCHIGNARD, M.D.
- Email: margaux.gaschignard@aphp.fr
- Phone: 01 44 49 40 23
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.