Qualitative Effects of AEF0217 in Down Syndrome People
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Down Syndrome (Trisomy 21). Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Spain
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Qualitative Evaluation of Perceived Effects Elicited by AEF0217 in Individuals With Down Syndrome
Overview
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.
Primary outcome measures
- - Overall, how would the caregiver describe the experience with the effect of the treatment the participant received during the trial? [Time frame: Day 1]
Eligibility criteria
Inclusion criteria
- 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)
Exclusion criteria
1- Not wanting to provide a written informed consent
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Other
Study locations
Spain · 1 center
- Hospital del Mar Research Institute — Barcelona
Identifiers
NCT: NCT07413224 · AEF0217-001