Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital
This study at Necker Hospital focuses on patients with inherited metabolic diseases to understand how consistently they take their daily oral medication. The main goal is to figure out if there's a need for a special educational workshop designed to help patients and their families stick to their treatment plans more effectively. Researchers will use a questionnaire and a ‘metabolic balance score’ to gather information. This research is important because taking medication regularly is key to managing these conditions, but there isn't much information on how well children and young people with metabolic diseases do this. By understanding the challenges, the study hopes to improve support for patients, ultimately leading to better health outcomes. It includes children from 7 years old to young adults up to 20 years old.
At a glance
What is this study about?
This study is being carried out at Necker Hospital and is looking at people who have inherited metabolic diseases. These are conditions where the body has trouble breaking down certain foods or chemicals. People with these conditions often need to take daily medication to stay healthy.
The main idea behind this study is to see how well patients, especially children and young adults aged 7 to 20, are taking their medicines as prescribed. Sometimes it can be tricky to remember to take pills every day, or to follow special diets. The researchers want to understand if there are common challenges that make it hard for patients to stick to their treatment plans.
By gathering this information, the hospital hopes to find out if there's a need for a special teaching programme or workshop. This workshop would be designed to help patients and their families learn practical ways to manage their daily medication and treatments more easily. Ultimately, the goal is to help people with these conditions stay healthier and feel better.
Key takeaways
- This study looks at how well patients with metabolic diseases take their daily oral medicines.
- It's for patients aged 7 to 20 who are seen at Necker Hospital.
- The goal is to see if a special workshop is needed to help patients manage their medication.
- Participation involves answering a questionnaire during a routine hospital visit.
- There are no new treatments or extra appointments involved.
Who may be eligible?
To join this study, you must be a patient who is already looked after by the Hereditary Metabolic Diseases department at Necker Hospital. You must be having a regular check-up there and also be taking a specific daily medication by mouth.
The study includes children who are at least 7 years old, and goes up to young adults who are 20 years old. If you are a child or teenager, your parents or guardians must agree for you to take part. Adults joining the study must agree themselves. Everyone involved must be able to understand and speak French.
You cannot join if your metabolic disease doesn't involve daily oral medication (for example, if you only have intravenous treatments or special diets). Also, if you or your parents do not speak French well enough, or if you simply don't want to take part, then you won't be included.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you between 7 and 20 years old?
- Are you a patient at the Hereditary Metabolic Diseases department at Necker Hospital?
- Do you take a specific daily medication by mouth for your metabolic condition?
- Can you (or your parents/guardians, if you are under 18) understand and speak French?
- Are you willing to answer some questions about your medication?
What does participation involve?
If you decide to take part in this study, it will involve answering a questionnaire during one of your usual check-up visits to the Hereditary Metabolic Diseases department at Necker Hospital. This questionnaire will ask questions about how you manage your medication and treatment. The study will also involve a doctor or nurse calculating a 'metabolic balance score' for you. This score helps them understand how well your metabolic condition is controlled. You will not need to take any new medicines or have extra hospital visits beyond your normal care. The information will be collected during one of your routine appointments.
Potential risks and benefits
Locations (1)
- Hôpital Necker-Enfants MaladesVerified postcodeParis, France· Recruiting
Common questions
What is a 'metabolic disease'?
Metabolic diseases are conditions where your body has trouble breaking down food or certain chemicals properly. This often means you need special diets or daily medicines.
What does 'treatment adherence' mean?
Treatment adherence means how well you follow your prescribed treatment plan, like remembering to take your medicines every day or sticking to a special diet.
Will I have to take any new medicines?
No, this study does not involve taking any new medicines. It's about how you manage your current daily oral medication.
What will I have to do if I join?
You will answer some questions in a questionnaire during one of your regular check-up appointments at Necker Hospital. A 'metabolic balance score' will also be calculated by your medical team.
Who is running this study?
This study is being run by the Department of Hereditary Metabolic Diseases at Necker Hospital.
How to find out more
Margaux GASCHIGNARD, M.D.
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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