AEF0217 in Participants With Down Syndrome

Summary

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

• Down Syndrome (Trisomy 21)

Interventions

DRUG

AEF0217 100 µg

Sachet of granules

DRUG

AEF0217 300 µg

sachet of granules

DRUG

Placebo

sachet of matching placebo granules

Sponsors & Collaborators

• European Commission

  collaborator OTHER

• BioClever 2005 S.L.

  collaborator OTHER

• Eurofins ADME, S.L.

  collaborator UNKNOWN

• Pharmalex

  collaborator UNKNOWN

• Hospital del Mar Research Institute (IMIM)

  collaborator OTHER

• Aelis Farma

  lead INDUSTRY

Principal Investigators

• Pier Vincenzo PIAZZA, MD, PhD · Aelis Farma

• Rafael DE LA TORRE FORNELL, PharmD, PhD · Hospital del Mar Research Institute

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

16 Years

Max Age

32 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-12-22

Primary Completion

2027-12-31

Completion

2027-12-31

Countries

• France
• Italy
• Spain

Study Locations