Down Syndrome (Trisomy 21)NCT07413224
Qualitative Effects of AEF0217 in Down Syndrome People
An observational study of Down Syndrome (Trisomy 21), run by Aelis Farma at 1 site in Spain.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Down Syndrome (Trisomy 21)
- How many people
- 28 participants
- Where
- 1 siteSpain
- Study dates
- 1 Mar 2026to May 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Caregivers and participants who have participated and completed the RCT AEF0217-201 (NCT 05748405)
Who may be able to join
- Participants having completed the trial AEF0217-102
- Being the caregiver of the participant
- Provided written informed consent (or assent in case of individuals with Down syndrome with no capacity to legally sign the consent)
Who cannot join
- 1- Not wanting to provide a written informed consent
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Qualitative Evaluation of Perceived Effects Elicited by AEF0217 in Individuals With Down Syndrome
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.
Read the detailed description
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.
- Conditions
- Down Syndrome (Trisomy 21)
- Medical subject headings
- Down Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Group AEF0217 | Participants who received AEF0217 in the previous trial AEF0217-102 | |
| Group Placebo | Participants who received Placebo in the previous trial AEF0217-102 |
What is being measured
The primary outcome is the main question the study is designed to answer.
- 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
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2026
- Study start1 Mar 2026
- Primary completionMay 2026 (estimated)
- Study completionMay 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Down Syndrome (Trisomy 21)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Aelis Farma
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Hospital del Mar Research Institute (IMIM)
- Responsible party
- Sponsor
- Investigators
- Pier Vincenzo Piazza, MD, PhD, Study director, Aelis Farma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07413224
- Sponsor's protocol ID
- AEF0217-001
- Status last verified
- Apr 2026
- First submitted
- 10 Feb 2026
- Last updated
- 8 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Qualitative Evaluation of Perceived Effects Elicited by AEF0217 in Individuals With Down Syndrome
- ClinicalTrials.gov ID
- NCT07413224
- Acronym / sponsor ID
- AEF0217-001
- Conditions
- Down Syndrome (Trisomy 21)
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 28 participants (estimated)
- Lead sponsor
- Aelis FarmaIndustry (pharma, biotech, devices)
- Collaborators
- Hospital del Mar Research Institute (IMIM)
- Study start
- 1 Mar 2026 (actual)
- Primary completion
- May 2026 (estimated)
- Study completion
- May 2026 (estimated)
- First posted
- 17 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group AEF0217 | – | –Participants who received AEF0217 in the previous trial AEF0217-102 |
| Group Placebo | – | –Participants who received Placebo in the previous trial AEF0217-102 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Caregivers and participants who have participated and completed the RCT AEF0217-201 (NCT 05748405)
Key inclusion criteria (3 listed)
- Participants having completed the trial AEF0217-102
- Being the caregiver of the participant
- Provided written informed consent (or assent in case of individuals with Down syndrome with no capacity to legally sign the consent)
Key exclusion criteria (1 listed)
- 1- Not wanting to provide a written informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
- Overall, how would the caregiver describe the experience with the effect of the treatment the participant received during the trial?
Time frame: Day 1
* 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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Pier Vincenzo Piazza, MD, PhD study director
Aelis Farma
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Down Syndrome (Trisomy 21), run by Aelis Farma at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 8 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
- Overall, how would the caregiver describe the experience with the effect of the treatment the participant received during the trial? (time frame: Day 1). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2026.