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| Name | Class |
|---|---|
| Hospital del Mar Research Institute (IMIM) | OTHER |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Group AEF0217 | Participants who received AEF0217 in the previous trial AEF0217-102 | ||
| Group Placebo | Participants who received Placebo in the previous trial AEF0217-102 |
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| Measure | Description | Time Frame |
|---|---|---|
| - Overall, how would the caregiver describe the experience with the effect of the treatment the participant received during the trial? |
| Day 1 |
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| Measure | Description | Time Frame |
|---|---|---|
| Overall, how the study satisfied the effects the caregiver was expecting in the participant after receiving the treatment?? |
| Day 1 |
Inclusion Criteria:
Exclusion Criteria:
1- Not wanting to provide a written informed consent
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Caregivers and participants who have participated and completed the RCT AEF0217-201 (NCT 05748405)
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| Name | Affiliation | Role |
|---|---|---|
| Pier Vincenzo Piazza, MD, PhD | Aelis Farma | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital del Mar Research Institute | Barcelona | 08003 | Spain |
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| ID | Term |
|---|---|
| D004314 | Down Syndrome |
| ID | Term |
|---|---|
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
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| Which treatment does the caregiver believe the participant received before knowing the actual assignment (active drug or placebo) |
|
| Day 1 |
| Was the caregiver's assumption about the treatment allocation correct? |
| 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:
Percentage calculated. - Completed by the caregiver | Day 1 |
| Ability to manage personal needs: eating, dressing, bathing, hygiene |
| Enrolment day |
| Ability to carry out household responsibilities, such as cleaning up, completing chores, and preparing food |
| Day 1 |
| Improvement in his/her engagement in structured activities or as a student, an apprentice or in his/her paid job |
| Day 1 |
| Improvement in his/her ability to adapt to new situations |
| Day 1 |
| Improvement in his/her ability to handle responsibilities |
| Day 1 |
| Improvement in his/her ability to face challenges and stress |
| Day 1 |
| Improvement in his/her ability to apply numerical concepts in everyday situations, such as understanding time, dates, and handling of money (cash) |
| Day 1 |
| Improvement his/her ability to navigate life outside the home, as for example have an improved safety awareness and use of transportation |
| Day 1 |
| Ability to relate and interact with others |
| Day 1 |
| Ability to maintain and develop relationships |
| Day 1 |
| He/she is more empathic |
| Day 1 |
| Increased activity in participating in recreational activities with others |
| Day 1 |
| Improvement in adapting to new various social situations |
| Day 1 |
| Ability to take part in a conversation/communication |
| Day 1 |
| He/she listens more attentively and/or responds more appropriately |
| Day 1 |
| Understand better spoken and nonverbal information |
| Day 1 |
| Can better express needs, thoughts, and emotions using verbal language |
| Day 1 |
| Write more often and/or use better grammar and/or spelling |
| Day 1 |
| Reads and better understand texts. |
| Day 1 |
| Thinking about the participant's abilities while taking the treatment, what changes (if any) did the caregiver notice? |
| 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? |
| 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 ? |
| Day 1 |
| Does the participant remember the effects of the treatment with AEF0217? |
| Day 1 |
| Did the participant notice any change to the better in abilities or behaviors after participating in the AEF0217 study? |
| Day 1 |
| If the participant observed changes, in which areas did they happen ? |
| Day 1 |
| Does the participant think the treatment helped daily life ? |
| Day 1 |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |