Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Down Syndrome clinical trials
Every US clinical trial registered for Down Syndrome — phase mix, recruiting status, and the sponsors running them, straight from the NIH ClinicalTrials.gov registry.
55 US clinical trials · 21 currently recruiting
The research picture
Down Syndrome has 55 registered US clinical trials, 21 of them open to new participants right now — about 38% of the total.
- 21
- recruiting participants now
- 38%
- of trials open to enrollment
- 5
- in Phase 3–4 (later-stage)
- 3
- top sponsor: Sequenom
Counts reflect the public ClinicalTrials.gov registry as last mirrored by PlainTrial. Status and phase are reported by each study's sponsor. This is reference information, not medical advice.
Active & Recent Trials
DS-Connect®: The Down Syndrome Registry
University of Colorado, Denver
NCT01950624
The Human Trisome Project
University of Colorado, Denver
NCT02864108
Specimen Collection from Pregnant Women At Increased Risk for Fetal Aneuploidy
Sequenom
NCT01429389
Patterns of Neurodevelopmental Disorders
Richard Frye
NCT06213090
Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy
National Institute of Allergy and Infectious Diseases (NIAID)
NCT07416201
A Study to Compare Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL)
National Cancer Institute (NCI)
NCT04546399
Trial-Ready Cohort-Down Syndrome (TRC-DS)
University of Southern California
NCT04165109
Physical Activity and Community EmPOWERment Project
University of North Carolina, Chapel Hill
NCT06740162
A Study to Learn More About the Health of Persons With Down Syndrome After Treatment for Acute Leukemia
Children's Oncology Group
NCT05702645
Treatment of Obstructive Sleep Apnea With Personalized Surgery in Children With Down Syndrome (TOPS-DS)
Oregon Health and Science University
NCT05508971
Down Syndrome Obstructive Sleep Apnea
Brigham and Women's Hospital
NCT06043440
Evaluating Treatment of ADHD in Children with Down Syndrome
Children's Hospital Medical Center, Cincinnati
NCT04219280
PAP for Children With DS and OSAS
University of Miami
NCT04132999
Brain Outcomes With Lifestyle Change in Down Syndrome
University of Kansas Medical Center
NCT05985486
Measuring Aided Language Development
University of Central Florida
NCT06512168
Gamma Frequency Stimulation in Individuals With Down Syndrome
Massachusetts Institute of Technology
NCT05196984
Self-Supporting Nasopharyngeal Airway (ssNPA) Treating Upper Airway Obstruction in Hypotonia
University of Michigan
NCT05527652
Medications for Obstructive Sleep Apnea to Improve Cognition in Children With Down Syndrome
University of Arizona
NCT05933603
How Simplified Language Affects Comprehension and Learning in Young Children With Down Syndrome
Michigan State University
NCT06951516
Sleep Intervention and Quality of Life in Down Syndrome
University of Alabama at Birmingham
NCT06783725
Harness-based Mobility Intervention for Infants With Down Syndrome
Boston University Charles River Campus
NCT06219863
Risk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic Lymphoma
Children's Oncology Group
NCT01190930
A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia
National Cancer Institute (NCI)
NCT03914625
Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome
Children's Oncology Group
NCT02521493
Evaluating Cognitive Outcomes in Down Syndrome
Children's Hospital Medical Center, Cincinnati
NCT04208685
Validation of Sleep Questionnaires in the Down Syndrome Population
Oregon Health and Science University
NCT03771469
Therapies for Down Syndrome Regression Disorder
University of Colorado, Denver
NCT05662228
Proactive Speech and Language Intervention for Infants With Down Syndrome
Arizona State University
NCT06450509
Investigating the Impact of JASPER Behavioral Therapy in Children With Down Syndrome
Boston Children's Hospital
NCT05638412
Effects of Hypoglossal Nerve Stimulation on Cognition and Language in Down Syndrome and Obstructive Sleep Apnea
Inspire Medical Systems
NCT04801771
Cholinergic Integrity in Down Syndrome in Association With Aging, Alzheimer's Disease Pathology, and Cognition
Vanderbilt University Medical Center
NCT05231798
Transcranial Photobiomodulation With Near-Infrared Light for Language in Individuals With Down Syndrome
Massachusetts General Hospital
NCT04668001
Clinical Evaluation of the SEQureDx T21 Test In High Risk Pregnancies
Sequenom
NCT01555346
Noninvasive Screening for Fetal Aneuploidy: A New Maternal Plasma Marker
Sequenom
NCT00971334
Expanded Noninvasive Genomic Medical Assessment: The Enigma Study
Progenity
NCT02787486
Congenital Heart Disease: Impact on Learning and Development in Down Syndrome (CHILD-DS)
Carelon Research
NCT05312177
Down Syndrome Metabolic Health Study
Children's Hospital of Philadelphia
NCT01821300
Down Syndrome Memantine Follow-up Study
University Hospitals Cleveland Medical Center
NCT02304302
A Bicycle Training Intervention
University of Michigan
NCT02046889
Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.
Life Molecular Imaging
NCT00928304
A Non-Drug Study of The Suitability of Neurocognitive Tests And Functioning Scales For The Measurement of Cognitive And Functioning Changes in Individuals With Down Syndrome
Hoffmann-La Roche
NCT01580384
Strategies for Teaching Verbs
Vanderbilt University Medical Center
NCT03441685
Neurocognitive and Psychosocial Outcomes in Survivors of Childhood Leukemia With Down Syndrome
St. Jude Children's Research Hospital
NCT02859389
A Pilot Study to Evaluate the Hypoglossal Nerve Stimulator in Adolescents With Down Syndrome and Obstructive Sleep Apnea
Christopher Hartnick, M.D.
NCT02344108
A Study of RG1662 in Individuals With Down Syndrome
Hoffmann-La Roche
NCT01436955
Oxygen Uptake Kinetics During Submaximal Exercise in Adults With Down Syndrome
University of Nevada, Las Vegas
NCT05995223
Medications for Obstructive Sleep Apnea In Children With Down Syndrome
University of Arizona
NCT04115878
Safety, Tolerability and Immunogenicity of ACI-24 Vaccine in Adults With Down Syndrome
AC Immune
NCT02738450
Relationship Between Down Syndrome (DS) and Alzheimer's Disease (AD)
St. Joseph's Hospital and Medical Center, Phoenix
NCT02759887
Investigating Trunk Control in Young Children With Down Syndrome
Texas Woman's University
NCT03544840
Parents as the Primary Sexuality Educators for Their Young Adults With Down Syndrome
Wake Forest University Health Sciences
NCT03135236
Treatment Trial of Subclinical Hypothyroidism in Down Syndrome
Children's Hospital of Philadelphia
NCT01832753
Down Syndrome Biomarker Initiative (DSBI)
Michael Rafii
NCT02141971
Balance Bike Training in Down Syndrome
University of Mississippi Medical Center
NCT03677245
Feasibility and Dose Tolerability of HD-tDCS in Healthy Adults and Children With Down Syndrome
University of Wisconsin, Madison
NCT02481765
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 2 |
| Phase 2 | 8 |
| Phase 3 | 4 |
| Phase 4 | 1 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Down Syndrome Trial Landscape
ClinicalTrials.gov lists 55 US studies indexed under Down Syndrome, and 21 of those are currently open to recruitment — roughly 38% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
The phase distribution for Down Syndrome shows 5 late-stage studies (Phase 3 and Phase 4 combined) alongside 10 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
Top sponsor activity for Down Syndrome is led by Sequenom with 3 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 55 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Down Syndrome?
PlainTrial tracks 55 US clinical trials for Down Syndrome, of which 21 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Down Syndrome?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology — how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.