AEF0217 in Participants With Down Syndrome
ConditionDown Syndrome (Trisomy 21)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age16-32
SponsorAelis Farma
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
1. Male and female. For males: Throughout the trial and until the end of the trial, male participants should refrain from donating sperm and, if sexually active, use double-barrier contraceptive methods (i.e., male condoms and spermicide), or the female partner must use the same highly effective contraceptive methods as female trial participants .
2. Age ≥16 to ≤32years.
3. BMI ≥18.0 and ≤35 kg/m2.
4. Clinical diagnosis of Down syndrome (full trisomy 21 or translocations) documented by chromosomal analysis (karyotyping).
Disqualifiers
1. Pregnant or nursing female. Down syndrome
2. Mosaic Down syndrome or Down Syndrome Regression Disorder (DSRD). Medical history and clinical status
3. 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.
4. 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.
Trial design
Design model
Parallel
Treatments tested in this trial
AEF0217 100 µg
DrugSachet of granules
AEF0217 300 µg
Drugsachet of granules
Placebo
Drugsachet of matching placebo granules
Treatment groups
188 Participants
are divided into 4 treatment groupsGroup A: AEF0217 0.1 mgExperimental treatment 2 interventions
Group B: AEF0217 0.2 mgExperimental treatment 1 intervention
Group C: AEF0217 0.6 mgExperimental treatment 1 intervention
Group D: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Change from baseline of the normalized scores of the 9 subdomains of the VABS-3 at week 24 (end of treatment)
Raw scores will be normalised to 100. Higher scores on the VABS-3 indicate better adaptive functioning.
Time frame
Baseline and Week 24Secondary outcomes
1
Changes from baseline in the fluid cognition composite change sensitive score of the NIH-ToolBox for Intellectual Deficiencies at Week 24 (end of treatment)
Higher scores on the NIH-TCB for ID indicate better performance
Time frame
Baseline and Week 242
Changes from baseline in the individual change sensitive scores of the Flanker inhibitory control & attention test used to measure fluid cognition in the NIH-TB for ID at week 24 (End of treatment).
Higher scores indicate better performance
Time frame
Baseline and Week 243
Changes from baseline in the individual change sensitive scores of the Picture Sequence Memory test used to measure fluid cognition in the NIH-TB for ID at week 24 (End of treatment).
Higher scores indicate better performance
Time frame
Baseline and Week 244
Changes from baseline in the individual change sensitive scores of the List Sorting Working Memory test used to measure fluid cognition in the NIH-TB for ID at week 24 (End of treatment).
Higher scores indicate better performance
Time frame
Baseline and Week 24Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Aelis Farma
Lead sponsor
European Commission
Collaborator
BioClever 2005 S.L.
Collaborator
Eurofins ADME, S.L.
Collaborator
Pharmalex
Collaborator
Hospital del Mar Research Institute (IMIM)
Collaborator
Source ClinicalTrials.gov