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PHASE 2B TRIAL OF AEF0217 IN PARTICIPANTS WITH DOWN SYNDROME
EU CTIS ID: 2025-521013-10-00
What this study is testing
Identify the endpoints and doses of AEF0217 administered for 24 weeks which show an improvement in adaptive behaviours compared to placebo, the dynamic of these effects and the influence of age group, degree of disability (moderate vs mild), APOE4 genotype and plasma concentrations of AEA and 2-AG at baseline.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 01. Male and female.
- 10. The parent or caregiver must be a constant and reliable informant with sufficient contact with the participant to have detailed knowledge of the participant’s adaptive functioning to be able to answer accurately the questions asked by a neuropsychologist at the assessments.
- 11. Vital signs, ECG , and safety laboratory3 parameters must be without clinically relevant abnormalities as per the judgement of the investigator, except for: - Stable type 1 or 2 diabetes provided the participant is monitored regularly prior to and during the trial to ensure adequate glucose control. - Hypothyroidism controlled by treatment so that the participant is euthyroid and T4 stable (range 77–155 nmol/L) for at least 6 weeks prior to randomization. Fluctuations in TSH up to a maximum of 10 mIU/L are allowed.
- 02. Age ≥16 to ≤32years.
- 03. BMI ≥18.0 and ≤35 kg/m2
- 04. Clinical diagnosis of Down syndrome (full trisomy 21 and translocations) documented by chromosomal analysis (karyotyping).
You likely can't join if
- 01. Pregnant or nursing female.
- 23. Administration of an investigational medicinal product, including AEF0217, within the last 3 months prior to randomization.
- 02. Mosaic Down syndrome or Down Syndrome Regression Disorder (DSRD).
- 03. Active or clinically relevant conditions that could, in the investigator’s judgment, affect absorption, distribution, or metabolism of the trial medication (e.g., inflammatory bowel disease, gastric or duodenal ulcers or severe lactose intolerance); controlled celiac disease is allowed.
- 04. Clinically relevant obstructive pulmonary disease or asthma that is untreated. Patients well-controlled by treatment (inhalation or oral) for at least 6 weeks prior to screening may be included if considered safe by the investigator
- 05. Known severe obstructive sleep apnoea or if the investigator thinks that the person should be referred for a diagnosis/treatment of obstructive sleep apnoea.
See the full eligibility criteria
Who can join
- 01. Male and female.
- 10. The parent or caregiver must be a constant and reliable informant with sufficient contact with the participant to have detailed knowledge of the participant’s adaptive functioning to be able to answer accurately the questions asked by a neuropsychologist at the assessments.
- 11. Vital signs, ECG , and safety laboratory3 parameters must be without clinically relevant abnormalities as per the judgement of the investigator, except for: - Stable type 1 or 2 diabetes provided the participant is monitored regularly prior to and during the trial to ensure adequate glucose control. - Hypothyroidism controlled by treatment so that the participant is euthyroid and T4 stable (range 77–155 nmol/L) for at least 6 weeks prior to randomization. Fluctuations in TSH up to a maximum of 10 mIU/L are allowed.
- 02. Age ≥16 to ≤32years.
- 03. BMI ≥18.0 and ≤35 kg/m2
- 04. Clinical diagnosis of Down syndrome (full trisomy 21 and translocations) documented by chromosomal analysis (karyotyping).
- 05. Must be independently mobile and have sufficient vision and hearing to participate in the trial evaluations.
- 06. IQ >35–70 measured with Leiter-3. Individuals with IQ from >35 to <40 must have adequate cognitive and behavioural abilities according to the judgment of the principal investigator.
- 07. VCI of WISC-V language test score ≥ 4, based on mental age (estimated via IQ).
- 08. Must be able to understand most of the time and to express if he/she does not understand to the extent that he/she can accept the trial procedures. Must not use other forms of communication, signs, symbol boards, or devices as his/her primary form of communication.
- 09. Must have a parent or other reliable caregiver who agrees to accompany the participant to all clinic visits, provide information about the participant as required by the protocol, and ensure compliance with the medication schedule and protocol requirements
What rules you out
- 01. Pregnant or nursing female.
- 23. Administration of an investigational medicinal product, including AEF0217, within the last 3 months prior to randomization.
- 02. Mosaic Down syndrome or Down Syndrome Regression Disorder (DSRD).
- 03. Active or clinically relevant conditions that could, in the investigator’s judgment, affect absorption, distribution, or metabolism of the trial medication (e.g., inflammatory bowel disease, gastric or duodenal ulcers or severe lactose intolerance); controlled celiac disease is allowed.
- 04. Clinically relevant obstructive pulmonary disease or asthma that is untreated. Patients well-controlled by treatment (inhalation or oral) for at least 6 weeks prior to screening may be included if considered safe by the investigator
- 05. Known severe obstructive sleep apnoea or if the investigator thinks that the person should be referred for a diagnosis/treatment of obstructive sleep apnoea.
- 06. Recent (≤1 year) or ongoing haematologic or oncologic disorders (mild anaemia is allowed).
- 07. History of active epilepsy, recurrent seizures, infantile spasms with ongoing neurological sequelae, severe head trauma, or central nervous system infections (e.g., meningitis). Participants with remote childhood febrile seizures or resolved infantile spasms without seizures for ≥10 years and without current anti-seizure treatment may be included after medical evaluation and medical monitor and /or sponsor confirmation.
- 08. Clinically relevant unstable gastrointestinal, renal, hepatic, endocrine (including metabolic syndrome), or cardiovascular system disease as per the investigator’s judgement.
- 09. Any prevailing psychiatric disorder diagnosed using the DSM-5 that dominates a person's overall clinical condition outside of Down syndrome. If symptoms consistent with a diagnosis of psychiatric illness are detected during the screening assessment (NPI-Q) and considered as dominating, the investigator may request a psychiatric evaluation. If necessary, a consultant psychiatrist will be responsible for the final diagnosis, as this is not the investigator’s responsibility.
- 16. Known hypersensitivity to AEF0217 or fructose intolerance.
- 10. Participants with secondary psychiatric disorders including conduct disorders, attention deficit hyperactivity disorder, depressive disorders, anxiety disorders, and others that: 1) dominate the overall clinical condition according to the investigator’s assessment; and/or 2) are not stabilized by medical or behavioural treatments, a stabilized treatment being defined as a stable therapeutic regimen and dose for the 3 months prior to randomization; and/or 3) the type of pharmacological treatment is on the list of drugs that are prohibited.
- 11. Symptoms of early dementia confirmed by the NTG-EDSD
- 12. Substance use disorder as defined by the DSM-5.
- 14. Current diagnosis of epilepsy.
- 15. A history of intentional self-harm or suicide attempts brought on by suicidal thoughts. Suicidal ideation in the 12 months before screening, even if there was no suicide attempt or intentional self-harm. Assessed using 3 distinct questions about suicidal behaviour, suicidal ideation, and any self-harming actions.
- 20. Treatment with 1st generation neuroleptic drugs currently or within 3 months prior to randomization and benzodiazepines currently or in the 4 weeks prior to baseline assessments.
- 21. Intake of products containing EGCG (e.g., TEAVIGO, Mega Green Tea Capsules Life Extension, or Font-UP Grand Fontaine Laboratories) currently or during the last 4 weeks prior to the baseline assessments.
- 22. Treatment with medications or very regular consumption of fruits (i.e., pomelo/grapefruit) or natural remedies (i.e., hypericum preparations) known to strongly or moderately induce or inhibit CYP3A4/5 P450 isozymes.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.