Down Syndrome (Trisomy 21)
Conditions
Brief 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. 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.
Interventions
None listed
Sponsors
Aelis Farma
Hospital del Mar Research Institute (IMIM)
Study design
Observational model
OTHER
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
1. Participants having completed the trial AEF0217-102 2. Being the caregiver of the participant 3. 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| - Overall, how would the caregiver describe the experience with the effect of the treatment the participant received during the trial? | 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 |
Countries
Spain
Contacts
STUDY_DIRECTORPier Vincenzo Piazza, MD, PhD
Aelis Farma
Outcome results
None listed