Qualitative Effects of AEF0217 in Down Syndrome People

NCT07413224 | Enrolling By Invitation

• 1 other identifier: AEF0217-001
• Study Type: observational
• Target: 28
• Locations: 1 country
• Sites: 1

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.

Conditions

Down Syndrome (Trisomy 21)

Outcome Measures

Primary Outcomes (1)

• - 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

  Day 1

Other Outcomes (30)

• Overall, how the study satisfied the effects the caregiver was expecting in the participant after receiving the treatment??

  Day 1

• 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

Study Arms (2)

Group AEF0217

Participants who received AEF0217 in the previous trial AEF0217-102

Group Placebo

Participants who received Placebo in the previous trial AEF0217-102

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Caregivers and participants who have participated and completed the RCT AEF0217-201 (NCT 05748405)

You may qualify if:

• 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)

You may not qualify if:

• Not wanting to provide a written informed consent

Sponsors & Collaborators

• Aelis Farma: lead
• Hospital del Mar Research Institute (IMIM): collaborator

Study Sites (1)

Hospital del Mar Research Institute

Barcelona, 08003, Spain

Location

MeSH Terms

Conditions

Down Syndrome

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn

Study Officials

• Pier Vincenzo Piazza, MD, PhD

  Aelis Farma

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 10, 2026
• First Posted: February 17, 2026
• Study Start: March 1, 2026
• Primary Completion: May 1, 2026
• Study Completion: May 1, 2026
• Last Updated: April 8, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)