Actively Recruiting
AEF0217 in Participants With Down Syndrome
Led by Aelis Farma · Updated on 2026-04-08
188
Participants Needed
10
Research Sites
105 weeks
Total Duration
On this page
Sponsors
A
Aelis Farma
Lead Sponsor
E
European Commission
Collaborating Sponsor
AI-Summary
What this Trial Is About
The goal of this clinical trial is to identify if AEF0217 show an improvement in adaptive behaviors (daily life activities) in adults and older adolescents with Down Syndrome. It will also learn about the safety of AEF0217. The main questions it aims to answer are: * Does AEF0217 improve the daily life activities of the participants after being administered daily for 24 weeks ? * Does AEF0217 improve fluid cognitive function (cognitive abilities that do not depend on prior knowledge) and the crystallised one (knowledge acquired through one's culture, including verbal ability and social knowledge), the quality of life and sleep of the participants after being administered daily for 24 weeks ? * What medical problems do participants have when taking AEF0217? Researchers will compare 3 doses of AEF0217 to a placebo (a look-alike substance that contains no drug) to see if AEF0217 improves adaptative behaviours in people with Down Syndrome. Participants will: * Take AEF0217 or a placebo every day for 24 weeks * Visit the clinic 6 times with their caregiver for checkups, performing tests on a tablet and answering questionnaires. * Be called by phone at home 5 times to check that they are well.
CONDITIONS
Official Title
AEF0217 in Participants With Down Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female participants aged 16 to 32 years
- Body mass index (BMI) between 18.0 and 35 kg/m2
- Clinical diagnosis of Down syndrome confirmed by chromosomal analysis
- Independently mobile with sufficient vision and hearing for study participation
- IQ between >35 and 70 measured by Leiter-3 test; those with IQ >35 to <40 must have adequate cognitive and behavioral abilities
- Verbal Comprehension Index (VCI) of WISC-V language test score >4 based on mental age
- Ability to understand and express needs during trial procedures without using alternative communication methods
- Have a parent or reliable caregiver who will attend all visits, provide information, and help ensure compliance
- Caregiver must have detailed knowledge of participant's adaptive functioning
- Vital signs, ECG, and lab tests without clinically relevant abnormalities, except controlled diabetes or hypothyroidism
- Assent by participant and consent by legally authorized representative, or participant consent if applicable
- Caregiver provides informed consent to fulfill caregiver responsibilities
You will not qualify if you...
- Pregnant or nursing females
- Mosaic Down syndrome or Down Syndrome Regression Disorder
- Active or clinically relevant conditions affecting drug absorption or metabolism (except controlled celiac disease)
- Untreated obstructive pulmonary disease or asthma; controlled cases may be included if safe
- Known severe obstructive sleep apnea or suspected untreated sleep apnea
- Recent or ongoing hematologic or oncologic disorders (mild anemia allowed)
- History of infantile spasms, epilepsy, severe head trauma, or CNS infections except isolated febrile seizures over 8 years ago
- Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular diseases
- Prevailing psychiatric disorders dominating clinical condition outside Down syndrome
- Secondary psychiatric disorders that dominate condition, are not stabilized, or require prohibited medications
- Symptoms of early dementia
- Substance use disorder
- Positive urine test for alcohol or drugs at screening
- Current epilepsy diagnosis
- History or recent suicidal behavior or ideation
- Hypersensitivity to AEF0217 or fructose intolerance
- Clinically significant illness or infection within 2 weeks before randomization
- Life-threatening diseases
- Other clinically significant diseases or conditions that may affect safety or trial conduct
- Recent use of first-generation neuroleptics or benzodiazepines
- Recent intake of products containing EGCG or certain foods or remedies affecting CYP3A4/5 enzymes
- Use of investigational products including AEF0217 within 3 months before randomization
AI-Screening
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Trial Site Locations
Total: 10 locations
1
CHU de Bordeaux
Bordeaux, France, 33000
Not Yet Recruiting
2
Genetics department, Hospices Civils de Lyon
Lyon, France, 69500
Not Yet Recruiting
3
Service Génétique Médicale, CHU de Montpellier
Montpellier, France, 34295
Actively Recruiting
4
Genetics department, Institut Jérôme Lejeune
Paris, France, 75725
Actively Recruiting
5
Service de Génétique, Chromosomique et Moléculaire, Chu de Saint Etienne
Saint-Etienne, France, 42270
Actively Recruiting
6
IRCCS Istituto Delle Scienze Neurologiche, Azienda Unita Sanitaria Locale Di Bologna
Bologna, Italy, 40139
Actively Recruiting
7
Centro di Medicina dell'Invecchiamento, Policlinico Universitario Agostino Gemelli IRCCS
Roma, Italy, 00168
Actively Recruiting
8
UOR of Neurofarmacology and Translational Neurosciences, Associazione Oasi Maria S.S.Onlus
Troina, Italy, 94018
Actively Recruiting
9
Integrative Pharmacology and Systems Neurosciences, Hospital del Mar Research Institute
Barcelona, Spain, 08003
Actively Recruiting
10
Servicio de Medicina Interna, Hospital Universitario De La Princesa
Madrid, Spain, 28006
Not Yet Recruiting
Research Team
S
Stéphanie Monlezun, Ph. D
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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