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Down Syndrome

14
Pipeline Programs
20
Companies
38
Clinical Trials
4 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
9
0
4
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
1100%
+ 40 programs with unclassified modality

Competitive Landscape

15 companies ranked by most advanced pipeline stage

Colorado Therapeutics
Colorado TherapeuticsCO - Louisville
10 programs
3
1
1
MemantinePhase 41 trial
Fear ResponsePhase 31 trial
LorazepamPhase 2
Sargramostim for InjectionPhase 21 trial
TofacitinibPhase 2Small Molecule1 trial
+5 more programs
Active Trials
NCT01950624Recruiting100,000Est. Jun 2035
NCT04208685Active Not Recruiting202Est. Jun 2026
NCT02421276Completed45Est. Feb 2018
+6 more trials
Pfizer
PfizerNEW YORK, NY
4 programs
2
2
AriceptPhase 3
AriceptPhase 3
Donepezil HClPhase 2
Donepezil hydrochloridePhase 2
Inspire Medical Systems
Inspire Medical SystemsMN - Golden Valley
1 program
1
Inspire Upper Airway StimulationPhase 31 trial
Active Trials
NCT04801771Active Not Recruiting57Est. Sep 2027
Aelis Farma
Aelis FarmaFrance - Bordeaux
3 programs
1
AEF0217Phase 24 trials
Bordeaux Maze TestN/A1 trial
ElectroencephalographyN/A1 trial
Active Trials
NCT04241042Unknown50Est. Apr 2020
NCT04791124Unknown48Est. Jun 2021
NCT07413224Enrolling By Invitation28Est. May 2026
+3 more trials
Angeles Therapeutics
Angeles TherapeuticsCA - Los Angeles
2 programs
2
LorazepamPhase 21 trial
OxygenPhase 21 trial
Active Trials
NCT05662228Active Not Recruiting66Est. Dec 2026
NCT06043440Recruiting230Est. Dec 2027
AC Immune
AC Immune1015 LAUSANNE, Switzerland
1 program
1
ACI-24Phase 22 trials
Active Trials
NCT04373616Withdrawn0Est. Oct 2024
NCT02738450Completed20Est. Jun 2020
Human BioSciences
Human BioSciencesWV - Martinsburg
7 programs
AAC Technology - Standard of CareN/A1 trial
DS-Connect®: The Down Syndrome RegistryN/A
Down Syndrome Metabolic Health StudyN/A1 trial
Gross motor intervention, gross and fine motor interventionN/A1 trial
LevothyroxineN/A1 trial
+2 more programs
Active Trials
NCT03764761Terminated14Est. Nov 2021
NCT01821300Completed257Est. Aug 2017
NCT05144373Completed51Est. Aug 2025
+2 more trials
Eisai
EisaiChina - Liaoning
5 programs
Donepezil HClPHASE_21 trial
Donepezil hydrochloridePHASE_21 trial
E2020-Donepezil hydrochloridePHASE_21 trial
AriceptPHASE_31 trial
AriceptPHASE_31 trial
Active Trials
NCT00570128Completed129Est. Sep 2008
NCT00675025Terminated117Est. Dec 2008
NCT02094053Completed36Est. Apr 2017
+2 more trials
Oregon Therapeutics
Oregon TherapeuticsFrance - Paris
2 programs
Enhanced Milieu teachingN/A1 trial
custom orthoticsN/A1 trial
Active Trials
NCT03935620Completed3Est. Dec 2021
NCT01390558Completed23Est. Aug 2018
Augustine Therapeutics
Augustine TherapeuticsBelgium - Leuven
1 program
Hip Helpers Home ProgramN/A1 trial
Active Trials
NCT04603352Completed17Est. Jun 2025
Sound Pharmaceuticals
1 program
Supramalleolar OrthosesN/A1 trial
Active Trials
NCT01598350Completed6Est. Jun 2011
Genome & Company
Genome & CompanyKorea - Suwon
1 program
The Experience of Caregivers of Children With Down SyndromeN/A1 trial
Active Trials
NCT00695695Completed546Est. Feb 2014
Genomics
GenomicsUK - Oxford
1 program
biospecimen collectionN/A1 trial
Active Trials
NCT02759887Completed19Est. Dec 2018
Opko Health
Opko HealthMIAMI, FL
1 program
ELND005PHASE_21 trial
Active Trials
NCT01791725Completed23Est. Jun 2014
Life Molecular Imaging
Life Molecular ImagingGermany - Berlin
1 program
FlorbetabenPHASE_21 trial
Active Trials
NCT00928304Completed109Est. Jan 2011

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Colorado TherapeuticsMemantine
Colorado TherapeuticsFear Response
Inspire Medical SystemsInspire Upper Airway Stimulation
EisaiAricept
EisaiAricept
Aelis FarmaAEF0217
Angeles TherapeuticsOxygen
Colorado TherapeuticsSargramostim for Injection
Angeles TherapeuticsLorazepam
AC ImmuneACI-24
Colorado TherapeuticsTofacitinib
EisaiE2020-Donepezil hydrochloride
Opko HealthELND005
Life Molecular ImagingFlorbetaben
EisaiDonepezil hydrochloride

Showing 15 of 38 trials with date data

Clinical Trials (38)

Total enrollment: 105,325 patients across 38 trials

Efficacy and Safety of Memantine Hydrochloride in Enhancing the Cognitive Abilities of Young Adults With Down Syndrome

Start: Jul 2008Est. completion: Aug 201142 patients
Phase 4Completed

Exploring Sympathetic Nervous System Function in Individuals With Down Syndrome

Start: Dec 2025Est. completion: Dec 2029200 patients
Phase 3Not Yet Recruiting
NCT04801771Inspire Medical SystemsInspire Upper Airway Stimulation

Effects of Hypoglossal Nerve Stimulation on Cognition and Language in Down Syndrome and Obstructive Sleep Apnea

Start: Jun 2021Est. completion: Sep 202757 patients
Phase 3Active Not Recruiting

Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 6 To 10

Start: Sep 2008Est. completion: Dec 20089 patients
Phase 3Terminated

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

AEF0217 in Participants With Down Syndrome

Start: Dec 2025Est. completion: Dec 2027188 patients
Phase 2Recruiting

Down Syndrome Obstructive Sleep Apnea

Start: Oct 2023Est. completion: Dec 2027230 patients
Phase 2Recruiting
NCT05482334Colorado TherapeuticsSargramostim for Injection

Trial to Evaluate Safety and Efficacy of GM-CSF /Sargramostim in Down Syndrome

Start: Oct 2023Est. completion: Sep 20260
Phase 2Withdrawn

Therapies for Down Syndrome Regression Disorder

Start: Jun 2023Est. completion: Dec 202666 patients
Phase 2Active Not Recruiting

A Study of ACI-24 in Adults With Down Syndrome

Start: Oct 2021Est. completion: Oct 20240
Phase 2Withdrawn

Tofacitinib for Immune Skin Conditions in Down Syndrome

Start: Oct 2020Est. completion: Oct 202447 patients
Phase 2Completed
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 201423 patients
Phase 2Completed

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

Start: Jun 2009Est. completion: Jan 2011109 patients
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
NCT00570128EisaiDonepezil HCl

Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (HCl) (Aricept) In Treating Cognitive Dysfunction Exhibited By Children With Down Syndrome

Start: Nov 2007Est. completion: Sep 2008129 patients
Phase 2Completed

Phase 1/2 Trial of AEF0217 in Participants With Down Syndrome

Start: Dec 2022Est. completion: Oct 202440 patients
Phase 1/2Completed

AEF0217 First-in-Human Phase I Study in 3 Parts (Single and Multiple Ascending Doses, and Food Effect) in Healthy Subjects.

Start: Oct 2021Est. completion: Jun 202268 patients
Phase 1Completed

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

Start: Mar 2016Est. completion: Jun 202020 patients
Phase 1Completed

Qualitative Effects of AEF0217 in Down Syndrome People

Start: Mar 2026Est. completion: May 202628 patients
N/AEnrolling By Invitation
NCT05144373Human BioSciencesGross motor intervention, gross and fine motor intervention

Role of Early Motor Experience in Infants With Down Syndrome

Start: Jan 2022Est. completion: Aug 202551 patients
N/ACompleted
NCT05020366Human BioSciencesPromoting Health through Parent Empowerment and the Activation of Routines

Promoting Health Through Parent Empowerment and the Activation of Routines (PrO-PEAR)

Start: Oct 2021Est. completion: May 202340 patients
N/ACompleted
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
NCT04791124Aelis FarmaElectroencephalography

Assessment of Neural Oscillations in Adult Subjects With Down Syndrome and Typically Developing Subjects in Resting State and While Conducting Cognitive Tasks

Start: Jan 2021Est. completion: Jun 202148 patients
N/AUnknown
NCT04398862Colorado TherapeuticsPulmonary Health in Children With Down Syndrome

Pulmonary Health in Children With Down Syndrome

Start: Aug 2020Est. completion: Dec 202475 patients
N/AUnknown
NCT03935620Oregon TherapeuticsEnhanced Milieu teaching

Delivering Early Intervention to Children With Down Syndrome

Start: Jun 2019Est. completion: Dec 20213 patients
N/ACompleted
NCT04241042Aelis FarmaBordeaux Maze Test

Clinical Validation of the Bordeaux Maze Test

Start: Apr 2019Est. completion: Apr 202050 patients
N/AUnknown
NCT04208685Colorado TherapeuticsEvaluating Cognitive Outcomes in Down Syndrome

Evaluating Cognitive Outcomes in Down Syndrome

Start: Jun 2018Est. completion: Jun 2026202 patients
N/AActive Not Recruiting
NCT03764761Human BioSciencesAAC Technology - Standard of Care

Storybook Reading in Individuals With Down Syndrome

Start: Apr 2018Est. completion: Nov 202114 patients
N/ATerminated
NCT02759887Genomicsbiospecimen collection

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

Start: Nov 2016Est. completion: Dec 201819 patients
N/ACompleted
NCT02864108Colorado TherapeuticsThe Human Trisome Project

The Human Trisome Project

Start: Jul 2016Est. completion: Jul 20362,500 patients
N/ARecruiting

Depressed AIRE Gene Expression Causes Immune Cell Dysfunction & Autoimmunity in Down Syndrome

Start: Oct 2015Est. completion: Feb 201845 patients
N/ACompleted
NCT01950624Colorado TherapeuticsDS-Connect®: The Down Syndrome Registry

DS-Connect®: The Down Syndrome Registry

Start: Sep 2013Est. completion: Jun 2035100,000 patients
N/ARecruiting
NCT01821300Human BioSciencesDown Syndrome Metabolic Health Study

Down Syndrome Metabolic Health Study

Start: Feb 2013Est. completion: Aug 2017257 patients
N/ACompleted

Treatment Trial of Subclinical Hypothyroidism in Down Syndrome

Start: Jan 2013Est. completion: Oct 201712 patients
N/ACompleted

Gait Parameters of Persons With Down Syndrome With and Without Orthotics Using GaitRite

Start: Jun 2011Est. completion: Aug 201823 patients
N/ACompleted
NCT01598350Sound PharmaceuticalsSupramalleolar Orthoses

Gait in Children With Down Syndrome While Wearing Orthoses

Start: Jan 2010Est. completion: Jun 20116 patients
N/ACompleted
NCT00695695Genome & CompanyThe Experience of Caregivers of Children With Down Syndrome

The Experience of Caregivers of Children With Down Syndrome

Start: May 2008Est. completion: Feb 2014546 patients
N/ACompleted

Phase Legend

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

Key Insights

4 late-stage (Phase 3) programs, potential near-term approvals
4 actively recruiting trials targeting 105,325 patients
20 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.