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NCT05748405: MDDS
Phase 1/2 Trial of AEF0217 in Participants With Down Syndrome
Phase 1, PHASE2 trial testing Placebo in Down Syndrome in 40 participants. Completed in 7 October 2024.
7 October 2024
Quick facts
| Lead sponsor | Aelis Farma |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | quadruple |
| Primary purpose | treatment |
| Enrollment | 40 |
| Start date | 15 December 2022 |
| Primary completion | 7 October 2024 |
| Estimated completion | 7 October 2024 |
| Sites | 3 locations across Spain |
Drugs / interventions tested
- Placebo
- AEF0217 — full drug profile →
Conditions studied
- Down Syndrome — all drugs for Down Syndrome →
Sponsor
Aelis Farma — full company profile →
Who can join
Adults 18 to 35, any sex, with Down Syndrome. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
AEF0217-102 clinical trial assesses the safety, tolerability, plasma exposure and preliminary indications of pharmacodynamic activity of AEF0217 in female and male adult participants with Down syndrome between 18 and 35 years old. The trial AEF0217-102 is a double-blind, randomized, placebo-controlled, multiple-dose, 4-week phase 1/2 study. After a screening period, the participant will be randomised and will take an oral dose of AEF0217 0.2mg or placebo once a day for 28 days.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
-
Pharmacological inhibition of the CB1 cannabinoid receptor restores abnormal brain mitochondrial CB1 receptor expression and rescues bioenergetic and cognitive defects in a female mouse model of Rett syndrome.
Cosentino L, Urbinati C, Lanzillotta C, De Rasmo D, et al · · 2024 · cited 9× · PMID 39300547 · DOI 10.1186/s13229-024-00617-1 -
Targeting dysregulated CB1 receptors in a Down syndrome mouse model improves neurological outcomes.
Vázquez-Oliver A, Pérez-García S, Romero-Pérez R, Pizarro N, et al · · 2025 · PMID 41257439 · DOI 10.1002/alz.70874
Verify or expand the search:
- PubMed search for NCT05748405
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of AEF0217
Trials testing the same drug.
- NCT05170737 — AEF0217 First-in-Human Phase I Study in 3 Parts (Single and Multiple Ascending Doses, and Food Effect) in Healthy Subjec · Phase 1 · completed
Other recruiting trials for Down Syndrome
Currently open trials in the same condition.
- NCT07416201 — Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy · recruiting
- NCT07484464 — Effects of the Otago Exercise Program on Balance, Endurance, and Motor Coordination in Children With Down Syndrome · NA · recruiting
- NCT07428863 — Effects of Jump Rope on Navicular Drop in Down Syndrome · NA · recruiting
- NCT07260136 — Cervical Spine Abnormalities in Down Syndrome · active not recruiting
- NCT07248449 — Physical Fitness in DS: A Comparison of Cuevas Medak and Rebound Exercises · NA · recruiting
Other Aelis Farma trials
Trials by the same sponsor.
- NCT07334912 — AEF0217 in Participants With Down Syndrome · Phase 2 · recruiting
- NCT05451017 — The Effect of Food on the Oral Bioavailability of AEF0117 in Healthy Volunteers · Phase 1 · terminated
- NCT05554926 — Study of [4-14C] AEF0117 Following a Single Oral Dose in Healthy Male Subjects · Phase 1 · completed
- NCT05322941 — Effect of AEF0117 on Treatment-seeking Patients With Cannabis Use Disorder (CUD) · Phase 2 · completed
- NCT05170737 — AEF0217 First-in-Human Phase I Study in 3 Parts (Single and Multiple Ascending Doses, and Food Effect) in Healthy Subjec · Phase 1 · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT05748405 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Aelis Farma
- Last refreshed: 10 October 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05748405.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing