An observational study in Down Syndrome (Trisomy 21), sponsored by Aelis Farma. Enrolling by invitation at 1 site in Spain. Per ClinicalTrials.gov, last updated 2026-04-08.
Sponsored by Aelis Farma · Observational
Following the AEF0217-102 trial (NCT05748405), which investigated the effects of AEF0217 versus placebo, participants with Down syndrome and their caregivers who took part in the trial are invited to share their perceptions of what they experienced or observed, in an exploratory manner.
This qualitative study collects information through a specific questionnaire developed for this purpose.
This is a non-interventional, follow-up, cross-sectional, mixed-methods (qualitative and quantitative) study.
Participants and their caregivers who will be enrolled are those who have received either AEF0217 or placebo and who have completed the previous trial.
The participant and their cargiver will be contacted prospectively to assess their interest in participating in the study.
The participant and their caregiver will sign an electronic informed consent form and will receive a link to a questionnaire developed using EU Survey to be completed.
Data will be collected at a single time point but may refer retrospectively to past experiences related to the previous trial. Data from all participants and caregivers who complete the questionnaire will be included in the analysis.
Given the observational and descriptive nature of the study, the quantitative analysis will be primarily descriptive.
The study will be conducted in accordance with the ethical principles derived from the Declaration of Helsinki (75th General Assembly, Helsinki, Finland, October 2024).
In addition, the study will be conducted in compliance with the protocol, Regulation (EU) 2016/679 (General Data Protection Regulation, GDPR), and the regulatory requirements applicable at the study site.
438 studies on the registry are indexed under Down Syndrome; 99 are open to participants now.
This study's planned enrollment of 28 is below the median of 158 across 145 observational studies indexed under Down Syndrome.
Browse Down Syndrome studies →Aelis Farma is the lead sponsor of 11 studies on the registry; 2 are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 1 (17%) have results posted.
Counted across the registry records on this site, refreshed daily.
Caregivers and participants who have participated and completed the RCT AEF0217-201 (NCT 05748405)
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
1- Not wanting to provide a written
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants who received AEF0217 in the previous trial AEF0217-102
Participants who received Placebo in the previous trial AEF0217-102
- Overall, how would the caregiver describe the experience with the effect of the treatment the participant received during the trial?
* Rating of the global perception of the treatment period (Likert scale). Scale in 5 points (Very positive, Somewhat positive, Neutral, Somewhat negative, Very negative). * Completed by the caregiver
Time frame: Day 1
Overall, how the study satisfied the effects the caregiver was expecting in the participant after receiving the treatment??
* Rating of the global perception of the treatment period (Likert scale). Scale in 5 points (Very positive, Somewhat positive, Neutral, Somewhat negative, Very negative) * Completed by the caregiver
Time frame: Day 1
Which treatment does the caregiver believe the participant received before knowing the actual assignment (active drug or placebo)
* Rating Scale in 3 points (AEF0217, Placebo, I am not sure) * completed by the caregiver
Time frame: Day 1
Was the caregiver's assumption about the treatment allocation correct?
* Rating Scale in 2 points Yes or No * Completed by the caregiver
Time frame: Day 1
If the caregiver noticed an improvement what type of changes did the caregiver observe after the end of the treatment period?
Rating as follows: * The improvement continued up to this day * The improvement continued for some time and then started to decline * The improvement remained stable up to this day * The improvement remained stable for some time and then started to decline * The improvement started to decline shortly after the end of the treatment * After the decline the function returned to and stabilized at the level prior to the study * After the decline the functioning becomes progressively worse than the level prior to the study. Percentage calculated. \- Completed by the caregiver
Time frame: Day 1
Ability to manage personal needs: eating, dressing, bathing, hygiene
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Enrolment day
Ability to carry out household responsibilities, such as cleaning up, completing chores, and preparing food
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Improvement in his/her engagement in structured activities or as a student, an apprentice or in his/her paid job
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Improvement in his/her ability to adapt to new situations
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Improvement in his/her ability to handle responsibilities
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Improvement in his/her ability to face challenges and stress
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Improvement in his/her ability to apply numerical concepts in everyday situations, such as understanding time, dates, and handling of money (cash)
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Improvement his/her ability to navigate life outside the home, as for example have an improved safety awareness and use of transportation
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Ability to relate and interact with others
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Ability to maintain and develop relationships
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
He/she is more empathic
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Increased activity in participating in recreational activities with others
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Improvement in adapting to new various social situations
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Ability to take part in a conversation/communication
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
He/she listens more attentively and/or responds more appropriately
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Understand better spoken and nonverbal information
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Can better express needs, thoughts, and emotions using verbal language
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Write more often and/or use better grammar and/or spelling
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Reads and better understand texts.
* Amplitude of the observed changes * Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement * Completed by the caregiver
Time frame: Day 1
Thinking about the participant's abilities while taking the treatment, what changes (if any) did the caregiver notice?
* Qualitative (open text) * Thematic analysis to identify perceived domains of change and salient themes * Completed by the caregiver
Time frame: Day 1
In the caregiver's opinion, what impact did the changes, if any, had on the participant's wellbeing and the day-to-day activities of the family?
* Qualitative (open text) * Qualitative coding will be performed independently by two researchers using NVivo 15 (or similar). Discrepancies will be resolved through consensus discussions. Themes will be validated through iterative review and triangulated with quantitative findings to ensure coherence. • Thematic analysis to identify perceived domains of change and salient themes * Completed by the caregiver
Time frame: Day 1
In the caregiver's opinion, what impact did the changes, if any, have on the participant's interaction with others in the social ?
* Qualitative (open text) * Thematic analysis to identify perceived domains of change and salient themes * Completed by the caregiver
Time frame: Day 1
Does the participant remember the effects of the treatment with AEF0217?
* Binary variable: Yes or no * Completed by the participant with Down Syndrome
Time frame: Day 1
Did the participant notice any change to the better in abilities or behaviors after participating in the AEF0217 study?
* Only if the question 28 is answered "Yes" * Rated using a 4 levels scale (Big changes, Some changes, No change, Worsening) * Completed by the participant with Down Syndrome
Time frame: Day 1
If the participant observed changes, in which areas did they happen ?
* Only if the question 29 is answered "big changes" or some changes" * Rated using a multiple-choice question (more than one answer is possible) * My abilities to understand * My ability to speak * My ability to write * My ability to concentrate * My ability to pay attention * My ability to control my behavior * My abilities to learn at school * My ability to take care of myself * My ability to interact with others * My ability to perform my daily abilities * My ability to execute my job * Completed by the participant with Down Syndrome
Time frame: Day 1
Does the participant think the treatment helped daily life ?
* Only if the question 29 is answered "big changes" or some changes" * Rated using a 4 levels scale (Yes, a lot/Yes, a little/Not really/I don't know) * Completed by the participant with Down Syndrome
Time frame: Day 1
Plan to share: No
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