NCT02738450: 3-Star

A Phase Ib Multi-Center, Double-Blind, Randomized, Placebo-Controlled Dose Escalation Study of the Safety, Tolerability and Immunogenicity of ACI-24 in Adults With Down Syndrome

Completed Phase 1 Results posted Last updated 21 September 2021

What this trial tests

Phase 1 trial testing ACI-24 low dose in Down Syndrome in 20 participants. Completed in 1 June 2020.

Timeline

1 March 2016

Primary endpoint
1 June 2020

1 June 2020

Quick facts

Lead sponsor	AC Immune SA
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	sequential
Masking	quadruple
Primary purpose	treatment
Enrollment	20
Start date	1 March 2016
Primary completion	1 June 2020
Estimated completion	1 June 2020
Sites	4 locations across United States

Drugs / interventions tested

• ACI-24 low dose — full drug profile →
• ACI-24 high dose — full drug profile →
• Placebo

Conditions studied

• Down Syndrome — all drugs for Down Syndrome →

Sponsor

AC Immune SA — full company profile →

Who can join

Adults 25 to 45, any sex, with Down Syndrome. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Antibody Titer (Serum Anti-Aβ1-42 Free IgG) - Mean Absolute Value
  Time frame: Values at baseline (week 0) and week 50 are reported
  All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. The measure is expressed in Arbitrary Units per mL (AU/mL). AU/mL in a sample is obtained by back-calculation towards the standard curve.

Sponsor's own description

The purpose of this study is to test in adults with Down Syndrome the safety, tolerability and immunogenicity of a vaccine, ACI-24.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. History and progress of hypotheses and clinical trials for Alzheimer's disease.
   Liu PP, Xie Y, Meng XY, Kang JS. · · 2019 · cited 412× · PMID 31637009 · DOI 10.1038/s41392-019-0063-8
2. Advances in progressive supranuclear palsy: new diagnostic criteria, biomarkers, and therapeutic approaches.
   Boxer AL, Yu JT, Golbe LI, Litvan I, et al · · 2017 · cited 339× · PMID 28653647 · DOI 10.1016/s1474-4422(17)30157-6
3. Alzheimer's disease associated with Down syndrome: a genetic form of dementia.
   Fortea J, Zaman SH, Hartley S, Rafii MS, et al · · 2021 · cited 284× · PMID 34687637 · DOI 10.1016/s1474-4422(21)00245-3
4. Exploring the Pathogenesis of Alzheimer Disease in Basal Forebrain Cholinergic Neurons: Converging Insights From Alternative Hypotheses.
   Chen XQ, Mobley WC. · · 2019 · cited 124× · PMID 31133787 · DOI 10.3389/fnins.2019.00446
5. Passive immunotherapies targeting Aβ and tau in Alzheimer's disease.
   Plotkin SS, Cashman NR. · · 2020 · cited 106× · PMID 32682954 · DOI 10.1016/j.nbd.2020.105010
6. Detection and treatment of Alzheimer's disease in its preclinical stage.
   Rafii MS, Aisen PS. · · 2023 · cited 104× · PMID 37202518 · DOI 10.1038/s43587-023-00410-4
7. Further understanding the connection between Alzheimer's disease and Down syndrome.
   Snyder HM, Bain LJ, Brickman AM, Carrillo MC, et al · · 2020 · cited 82× · PMID 32544310 · DOI 10.1002/alz.12112
8. Safety, Tolerability, and Immunogenicity of the ACI-24 Vaccine in Adults With Down Syndrome: A Phase 1b Randomized Clinical Trial.
   Rafii MS, Sol O, Mobley WC, Delpretti S, et al · · 2022 · cited 33× · PMID 35532913 · DOI 10.1001/jamaneurol.2022.0983