Aelis Farma has launched its Phase IIb clinical trial evaluating AEF0217, targeting behavioural and cognitive impairments in individuals with Down syndrome (Trisomy 21).
Recruitment for the trial is currently underway across France, Italy, and Spain.
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The placebo-controlled, double-blind, parallel group, randomised, international multi-centre trial follows initial regulatory approvals and the first patient’s first visit in December 2025.
Submitted via the EU Clinical Trials Information System (CTIS), the study protocol received approval from the European Medicines Agency (EMA) and national authorities in France, Italy, and Spain under Regulation (EU) No 536/2014.
Currently, people with Down syndrome often experience persistent impairments in adaptive behaviour and cognition. No pharmacological therapies are approved for these symptoms; existing care relies on educational, behavioural, and supportive interventions.
In an earlier Phase I/II double-blind, placebo-controlled study involving 29 young adults with Down syndrome, AEF0217 demonstrated a favourable safety profile and statistically significant improvements in adaptive behaviour measured by Vineland Adaptive Behaviour Scales (VABS), alongside improved brain activity.
The latest study intends to enrol 188 participants aged 16 to 32 years across ten sites (five in France, three in Italy, two in Spain). It will allocate participants in a 1:1:1:1 ratio to receive oral AEF0217 at three dose levels or placebo for 24 weeks, followed by eight weeks of monitoring.
Primary endpoint analysis will assess change in adaptive behaviours after six months using VABS-3 scores; secondary endpoints include quality of life, sleep efficiency, and cognition. Interim safety data will be reviewed after 40 participants complete treatment for 12 weeks.
Recruitment is anticipated to be concluded by late 2026, with initial findings planned for the second half of 2027.
Aelis Farma CEO Pier Vincenzo Piazza said: “The successful start of recruitment of our Phase IIb trial marks an important milestone in the clinical development of AEF0217 and is a concrete demonstration that Aelis Farma is executing its roadmap with operational efficiency.
“Our collective ambition is clear: develop the first treatment able to improve the quality of life and independence of people with Down syndrome.”
