Down Syndrome Clinical Trials

72 recruitingLast updated: June 18, 2026

There are 72 actively recruiting down syndrome clinical trials across 24 countries. Studies span Not Applicable, Phase 2, Phase 3, Phase 1, Phase 4. Top locations include Lahore, Punjab Province, Pakistan, Aurora, Colorado, United States, Phoenix, Arizona, United States. Updated daily from ClinicalTrials.gov.


Down Syndrome Trials at a Glance

72 actively recruiting trials for down syndrome are listed on ClinicalTrialsFinder across 6 cities in 24 countries. The largest study group is Not Applicable with 36 trials, with the heaviest enrollment activity in Lahore, Aurora, and Phoenix. Lead sponsors running down syndrome studies include Riphah International University, University of Colorado, Denver, and Institut Jerome Lejeune.

Browse down syndrome trials by phase

Treatments under study

About Down Syndrome Clinical Trials

Looking for clinical trials for Down Syndrome? There are currently 62 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 72 trials

Recruiting
Phase 2

A Study to Compare Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL)

Recurrent B Acute Lymphoblastic LeukemiaDown Syndrome
National Cancer Institute (NCI)461 enrolled225 locationsNCT04546399
Recruiting
Not Applicable

How Simplified Language Affects Comprehension and Learning in Young Children With Down Syndrome

Down Syndrome
Michigan State University30 enrolled1 locationNCT06951516
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

The ADVANCE (Assay Development and Validation for Pre-Natal and Obstetric Conditions) Study is the Largest U.S.-Based Prospective Study Demonstrating a Circulating Fetal Cell (CFC) Based Approach to Non-invasive Fetal Risk Assessment

AneuploidyDown Syndrome (Trisomy 21)22q11.2 Deletion Syndrome+4 more
BillionToOne Inc.1,000 enrolled6 locationsNCT07643896
Recruiting
Not Applicable

tDCS for Catatonic Depression in Down Syndrome: A Pilot Study

Down Syndrome
Hôpital le Vinatier62 enrolled1 locationNCT06866925
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

Vascular Function in Adults With Down Syndrome

Down Syndrome (Trisomy 21)
University of Nevada, Las Vegas24 enrolled1 locationNCT07630207
Recruiting

Collaboration for Down Syndrome Progress (CDP)

Down SyndromeIntellectual and Developmental DisabilitiesChromosome Disorders
RTI International1,400 enrolled16 locationsNCT07625579
Recruiting
Phase 2

Cholinergic Integrity in Down Syndrome in Association With Aging, Alzheimer's Disease Pathology, and Cognition

Alzheimer DiseaseDown SyndromeDown Syndrome, Partial Trisomy 21
Vanderbilt University Medical Center30 enrolled1 locationNCT05231798
Recruiting
Not Applicable

Drumming Lessons' Influence on Children With Down Syndrome

Down Syndrome
Vanderbilt University30 enrolled1 locationNCT07156318
Recruiting

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

DementiaAlzheimer DiseaseDown Syndrome
University of Southern California550 enrolled22 locationsNCT04165109
Recruiting
Phase 2

Modulation of the Immune System in Down Syndrome for Improved Outcomes and Neurodevelopment - 1

Down Syndrome
University of Colorado, Denver92 enrolled1 locationNCT07598643
Recruiting
Not Applicable

Adapting RDAD for DS

Down Syndrome (DS)Alzheimer's Disease(AD)
University of Kansas Medical Center40 enrolled1 locationNCT07425769
Recruiting
Not Applicable

Effects of Bean Bag Tossing Game on Balance and Gait in Children With Down Syndrome.

Down Syndrome
Riphah International University36 enrolled1 locationNCT07578922
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
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 enrolled183 locationsNCT04726241
Recruiting
Phase 3

Exploring Sympathetic Nervous System Function in Individuals With Down Syndrome

Autonomic DysfunctionDown Syndrome
University of Colorado, Denver200 enrolled1 locationNCT07238465
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