AEF0217 in Participants With Down Syndrome
This research study is testing a new medication called AEF0217 for individuals with Down Syndrome, specifically those aged 16 to 32. The main goals are to find out if AEF0217 can improve daily life skills and thinking abilities, as well as to check if it's safe to use. Participants will be randomly assigned to take one of two doses of AEF0217 or a placebo (a dummy pill with no active drug) every day for 24 weeks. Researchers will closely monitor their progress through clinic visits, tests, and phone calls. This study takes place in clinics across Spain, France, and Italy and involves about 188 people.
At a glance
What is this study about?
You might be interested in a research study looking at a new medication called AEF0217 for people with Down Syndrome. This study is designed to explore whether AEF0217 can help improve daily life activities and thinking skills in adults and older teenagers with Down Syndrome. It's a 'Phase 2' study, which means researchers are trying to get a better understanding of the drug's effects and safety in a larger group of people, after earlier smaller studies have suggested it might be helpful.
The main questions this study hopes to answer are: Can AEF0217 improve how people with Down Syndrome manage their daily tasks after taking it for 24 weeks? Does it also help with different types of thinking skills, improve quality of life, and sleep? And importantly, what are the possible side effects or medical problems that people might experience when taking AEF0217? By answering these questions, researchers hope to learn if AEF0217 could be a useful new treatment option in the future.
To ensure fair results, some participants will receive the actual medication, while others will receive a 'placebo'. A placebo looks exactly like the study drug but contains no active medicine. This allows researchers to compare the effects of the active drug against no treatment. No one, not even the doctors or nurses, will know who is receiving which until the end of the study. This is called a 'double-blind' study and helps ensure the results are as accurate as possible.
Key takeaways
- Tests a new medication (AEF0217) for Down Syndrome.
- Aims to improve daily activities and thinking skills.
- Involves taking the drug or a dummy pill for 24 weeks.
- Includes regular clinic visits and phone calls.
- Participation is voluntary and you can leave at any time.
- Takes place in clinics across Spain, France, and Italy.
Who may be eligible?
To join this study, people need to be between 16 and 32 years old and have a confirmed diagnosis of Down Syndrome. They should be able to move around on their own and have good enough sight and hearing to take part in the tests. The study also requires participants to have a certain level of understanding and communication skills, which will be checked by the study team.
It's important that participants have a parent or strong caregiver who can come to all the clinic visits with them. This caregiver will also help make sure the participant takes their study medicine as agreed and provides information about how they are doing. This ensures consistent care and accurate feedback for the research team.
For safety, both male and female participants will need to follow specific guidelines regarding contraception throughout the study. This is common in drug trials to prevent any potential risks to unborn babies. The study team will explain these rules clearly if you are considering joining.
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 16 and 32 years old?
- Do you have a diagnosis of Down Syndrome?
- Can you move around on your own?
- Do you have a parent or caregiver who can attend all appointments with you?
- Are you able to understand and follow instructions for the study?
What does participation involve?
If you decide to take part, you will take either AEF0217 or a dummy pill every day for 24 weeks. Throughout this time, you will visit the clinic 6 times with your caregiver for check-ups, where you'll do tests on a tablet and answer questions. These visits include an initial check, three visits during the treatment period (at weeks 4, 12, and 24), and a final follow-up visit 8 weeks after you stop taking the study medication.
In between clinic visits, you'll receive 5 phone calls at home to check how you're feeling and if everything is going well. During these visits and calls, the study team will monitor your progress, safety, and how well the treatment might be working. This includes looking at your daily activities, thinking skills, quality of life, and sleep. We will also take blood and urine samples to check for any changes.
After completing the 24 weeks of treatment, and if it's approved, you might be invited to continue into an extra 12-month study where everyone gets the active drug. If you don't wish to join this extension, you will have a final follow-up 8 weeks after your treatment ends. The total time you'd be involved in the study, from start to finish, would be about 36 weeks.
Potential risks and benefits
Locations (10)
- CHU de BordeauxVerified postcodeBordeaux, France· Not yet recruiting
- Genetics department, Hospices Civils de LyonVerified postcodeLyon, France· Not yet recruiting
- Service Génétique Médicale, CHU de MontpellierVerified postcodeMontpellier, France· Recruiting
- Genetics department, Institut Jérôme LejeuneVerified postcodeParis, France· Recruiting
- Service de Génétique, Chromosomique et Moléculaire, Chu de Saint EtienneVerified postcodeSaint-Etienne, France· Recruiting
- IRCCS Istituto Delle Scienze Neurologiche, Azienda Unita Sanitaria Locale Di BolognaVerified postcodeBologna, Italy· Recruiting
- Centro di Medicina dell'Invecchiamento, Policlinico Universitario Agostino Gemelli IRCCSVerified postcodeRoma, Italy· Recruiting
- UOR of Neurofarmacology and Translational Neurosciences, Associazione Oasi Maria S.S.OnlusVerified postcodeTroina, Italy· Recruiting
- Integrative Pharmacology and Systems Neurosciences, Hospital del Mar Research InstituteVerified postcodeBarcelona, Spain· Recruiting
- Servicio de Medicina Interna, Hospital Universitario De La PrincesaVerified postcodeMadrid, Spain· Not yet recruiting
Common questions
What is AEF0217?
AEF0217 is a new medication being tested to see if it can help people with Down Syndrome with their daily activities and thinking skills.
What is a 'placebo'?
A placebo is a dummy pill that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers compare the effects of the active medicine against no treatment.
How long will I be in the study?
The study will last about 36 weeks in total. This includes 24 weeks of taking the study medicine and some follow-up appointments afterwards.
What if I want to stop taking part?
You are free to leave the study at any time, for any reason, without it affecting your medical care.
Where does the study take place?
This study is happening in several medical centres across Spain, France, and Italy.
How to find out more
Stéphanie Monlezun, Ph. D
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.