Actively Recruiting
Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital
Led by Assistance Publique - Hôpitaux de Paris · Updated on 2026-03-10
200
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
A
Assistance Publique - Hôpitaux de Paris
Lead Sponsor
U
URC-CIC Paris Descartes Necker Cochin
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating treatment adherence among children, adolescents, and young adults with inherited metabolic diseases who are followed at the Necker Hospital. The study aims to understand how well patients follow their medication regimens and to determine if a therapeutic education workshop focusing on medication adherence is needed. Previous studies have shown that adherence can decline during adolescence and is crucial for disease control in metabolic disorders. Participants include patients aged 7 to 20 years who are taking specific daily oral medications for their metabolic condition. During the study visit, patients and their parents will complete a pseudonymized questionnaire assessing treatment adherence, knowledge of the disease, and understanding of their medications. For patients with urea cycle deficiency, a metabolic balance score will also be calculated using medical records from the previous two years. Throughout the study, researchers will gather information on treatment adherence and correlate it with factors such as age, medication knowledge, and metabolic balance. The questionnaire responses will be collected during routine visits and will not be shared with treating physicians. The study will measure adherence initially at the time of the questionnaire, with participation lasting for the duration of the routine care visit.
CONDITIONS
Brief Title
Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients followed in the Hereditary Metabolic Diseases department of Necker Hospital during their usual care visit
- Children aged 7 years and older, adolescents, or young adults up to 20 years
- Taking a specific daily oral medication for their metabolic disease
- Patients and their parents or legal guardians must be informed and consenting to participate
You will not qualify if you...
- Patients with metabolic diseases not treated by oral medications (e.g., intravenous treatments, amino acid mixtures, dietary regimens)
- Patients and parents who are not proficient in French
- Refusal to participate by the patient or their legal guardians
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) during usual care
Duration - Single timepoint at study entry
Participants complete a questionnaire assessing treatment adherence, understanding of the illness, knowledge of medications, and number of oral medications taken during a follow-up visit. For patients with urea cycle deficiency, metabolic balance scores are calculated from medical records.
1 follow-up visit (in-person) during usual care
Trial Site Locations
Total: 1 location
1
Hôpital Necker-Enfants Malades
Paris, France, 75015
Actively Recruiting
Research Team
M
Margaux GASCHIGNARD, M.D.
H
Hélène Morel
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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