Down Syndrome Clinical Trials

54 recruiting

Down Syndrome Trials at a Glance

59 actively recruiting trials for down syndrome are listed on ClinicalTrialsFinder across 6 cities in 22 countries. The largest study group is Not Applicable with 32 trials, with the heaviest enrollment activity in Lahore, Cincinnati, and Phoenix. Lead sponsors running down syndrome studies include Riphah International University, Institut Jerome Lejeune, and University of Arizona.

Browse down syndrome trials by phase

Treatments under study

About Down Syndrome Clinical Trials

Looking for clinical trials for Down Syndrome? There are currently 54 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Down Syndrome trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Down Syndrome clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 59 trials

Recruiting

Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy

Down Syndrome
National Institute of Allergy and Infectious Diseases (NIAID)700 enrolled1 locationNCT07416201
Recruiting

Patterns of Neurodevelopmental Disorders

EpilepsyAutism Spectrum DisorderNeurodevelopmental Disorders+5 more
Richard Frye1,000 enrolled1 locationNCT06213090
Recruiting
Phase 2

AEF0217 in Participants With Down Syndrome

Down Syndrome (Trisomy 21)
Aelis Farma188 enrolled10 locationsNCT07334912
Recruiting

Oro-myofunctional Characteristics and Obstructive Sleep Apnea in Infants With Down Syndrome

Obstructive Sleep ApneaDown Syndrome (Trisomy 21)
Hospices Civils de Lyon30 enrolled1 locationNCT07195253
Recruiting
Phase 1Phase 2

The Pediatric Acute Leukemia (PedAL) Screening Trial - A Study to Test Bone Marrow and Blood in Children With Leukemia That Has Come Back After Treatment or Is Difficult to Treat - A Leukemia & Lymphoma Society and Children's Oncology Group Study

Acute Lymphoblastic LeukemiaAcute Myeloid LeukemiaMyelodysplastic Syndrome+5 more
PedAL BCU, LLC960 enrolled182 locationsNCT04726241
Recruiting
Phase 4

Endotype DIrected Treatment for OSA in Down Syndrome

Down SyndromeObstructive Sleep Apnea (OSA)
University of Arizona200 enrolled5 locationsNCT07280468
Recruiting

Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)

Heart FailurePainInsomnia+22 more
Duke University5,000 enrolled51 locationsNCT04278404
Recruiting

Trial-Ready Cohort-Down Syndrome (TRC-DS)

DementiaAlzheimer DiseaseDown Syndrome
University of Southern California550 enrolled22 locationsNCT04165109
Recruiting
Not Applicable

Gamma Frequency Stimulation in Individuals With Down Syndrome

Down Syndrome
Massachusetts Institute of Technology60 enrolled1 locationNCT05196984
Recruiting
Not Applicable

HomeGrown: A Family-based Lifestyle Intervention to Support Healthy Development of Young Children With Down Syndrome

Down SyndromeChild Obesity
UNC Lineberger Comprehensive Cancer Center38 enrolled1 locationNCT07296861
Recruiting
Not Applicable

Sleep Intervention and Quality of Life in Down Syndrome

Down SyndromeDown Syndrome (Trisomy 21)
University of Alabama at Birmingham20 enrolled1 locationNCT06783725
Recruiting
Not Applicable

Effects of the Otago Exercise Program on Balance, Endurance, and Motor Coordination in Children With Down Syndrome

Down Syndrome
Riphah International University30 enrolled1 locationNCT07484464
Recruiting
Not Applicable

Self-Supporting Nasopharyngeal Airway (ssNPA) Treating Upper Airway Obstruction in Hypotonia

Obstructive Sleep ApneaDown SyndromeTolerance+3 more
University of Michigan40 enrolled1 locationNCT05527652
Recruiting
Not Applicable

Brain Outcomes With Lifestyle Change in Down Syndrome

ObesityAlzheimer DiseaseDown Syndrome
University of Kansas Medical Center81 enrolled1 locationNCT05985486
Recruiting
Not Applicable

Effects of Jump Rope on Navicular Drop in Down Syndrome

Down Syndrome
Riphah International University32 enrolled1 locationNCT07428863
Recruiting

A Study to Learn More About the Health of Persons With Down Syndrome After Treatment for Acute Leukemia

B Acute Lymphoblastic Leukemia Associated With Down SyndromeDown SyndromeMyeloid Leukemia Associated With Down Syndrome
Children's Oncology Group330 enrolled70 locationsNCT05702645
Recruiting
Not Applicable

Physical Activity and Community EmPOWERment Project

Williams SyndromeWAGR SyndromePrader-Willi Syndrome+10 more
University of North Carolina, Chapel Hill376 enrolled2 locationsNCT06740162
Recruiting
Not Applicable

Treatment of Obstructive Sleep Apnea With Personalized Surgery in Children With Down Syndrome (TOPS-DS)

Obstructive Sleep ApneaDown Syndrome
Oregon Health and Science University303 enrolled7 locationsNCT05508971
Recruiting
Phase 3

Compassionate Use Study of Epi-ON Corneal Collagen Crosslinking Performed Using UVA Exposure on Eyes With Ectatic Corneal Diseases for Subjects With Down Syndrome

Down Syndrome (DS)KeratoconusForme Fruste Keratoconus (FFK)+1 more
Woolfson Eye Institute225 enrolled12 locationsNCT07135167
Recruiting

Immune Profile and Respiratory Parameters in Down Syndrome Children With Acute Pneumonia: Pre-Intervention Analysis

Down Syndrome
Badr University40 enrolled2 locationsNCT07366593