Down Syndrome

9
Pipeline Programs
8
Companies
9
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
6
0
2
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

6 companies ranked by most advanced pipeline stage

E
EisaiChina - Liaoning
5 programs
3
2
AriceptPhase 3
AriceptPhase 31 trial
Donepezil HClPhase 2
Donepezil hydrochloridePhase 21 trial
E2020-Donepezil hydrochloridePhase 21 trial
Active Trials
NCT00675025Terminated117Est. Dec 2008
NCT02094053Completed36Est. Apr 2017
NCT00754052Terminated8Est. Dec 2008
AC Immune
AC Immune1015 LAUSANNE, Switzerland
2 programs
1
1
ACI-24Phase 21 trial
ACI-24 low dosePhase 11 trial
Active Trials
NCT02738450CompletedEst. Jun 2020
NCT04373616WithdrawnEst. Oct 2024
Opko Health
Opko HealthMIAMI, FL
1 program
1
ELND005Phase 21 trial
Active Trials
NCT01791725CompletedEst. Jun 2014
Life Molecular Imaging
Life Molecular ImagingGermany - Berlin
1 program
1
FlorbetabenPhase 21 trial
Active Trials
NCT00928304CompletedEst. Jan 2011
Augustine Therapeutics
Augustine TherapeuticsBelgium - Leuven
1 program
Hip Helpers home programN/A1 trial
Active Trials
NCT04603352Completed17Est. Jun 2025
Genomics
GenomicsUK - Oxford
1 program
biospecimen collectionN/A1 trial
Active Trials
NCT02759887Completed19Est. Dec 2018

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
EisaiAricept
AC ImmuneACI-24
EisaiE2020-Donepezil hydrochloride
Opko HealthELND005
Life Molecular ImagingFlorbetaben
EisaiDonepezil hydrochloride
AC ImmuneACI-24 low dose
Augustine TherapeuticsHip Helpers home program
Genomicsbiospecimen collection

Clinical Trials (9)

Total enrollment: 197 patients across 9 trials

Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 11 To 17

Start: Sep 2008Est. completion: Dec 20088 patients
Phase 3Terminated

A Study of ACI-24 in Adults With Down Syndrome

Start: Oct 2021Est. completion: Oct 2024
Phase 2Withdrawn
NCT02094053EisaiE2020-Donepezil hydrochloride

A Double-blind, Placebo-controlled Comparative Study and Open-label Extension Study to Confirm the Efficacy and Safety of E2020 in Subjects With Down Syndrome Having Regression Symptoms and Disabled Activities of Daily Living.

Start: Sep 2013Est. completion: Apr 201736 patients
Phase 2Completed

A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia

Start: Sep 2013Est. completion: Jun 2014
Phase 2Completed

Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.

Start: Jun 2009Est. completion: Jan 2011
Phase 2Completed
NCT00675025EisaiDonepezil hydrochloride

Evaluating The Safety Of Donepezil Hydrochloride (Aricept) For Up To 1 Year In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome - Follow-Up To A 10-Week, Double-Blind, Placebo-Controlled Trial

Start: Apr 2008Est. completion: Dec 2008117 patients
Phase 2Terminated
NCT02738450AC ImmuneACI-24 low dose

Safety, Tolerability and Immunogenicity of ACI-24 Vaccine in Adults With Down Syndrome

Start: Mar 2016Est. completion: Jun 2020
Phase 1Completed
NCT04603352Augustine TherapeuticsHip Helpers home program

Impact of an Orthotic Garment on Gross Motor Skills for Infants With Down Syndrome

Start: May 2021Est. completion: Jun 202517 patients
N/ACompleted
NCT02759887Genomicsbiospecimen collection

Relationship Between Down Syndrome (DS) and Alzheimer's Disease (AD)

Start: Nov 2016Est. completion: Dec 201819 patients
N/ACompleted

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs — potential near-term approvals
8 companies competing in this space