Down Syndrome Clinical Trials

12 recruiting

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 114 of 14 trials

Recruiting
Not Applicable

Brain Outcomes With Lifestyle Change in Down Syndrome

ObesityAlzheimer DiseaseDown Syndrome
University of Kansas Medical Center81 enrolled1 locationNCT05985486
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
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

Retinal Imaging in Neurodegenerative Disease

Traumatic Brain InjuryHuntington DiseaseNeuro-Degenerative Disease+12 more
Duke University2,000 enrolled1 locationNCT03233646
Recruiting
Phase 3

LEvetiracetam to Prevent Seizures in Symptomatic Alzheimer's Disease in Adults With Down Syndrome

EpilepsyAlzheimer DiseaseSeizures+7 more
Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau120 enrolled5 locationsNCT07234695
Recruiting
Phase 1

Leucettinib-21 First-in-Human Phase 1 in Healthy Volunteers and Subjects With Down Syndrome and Alzheimer's Disease

Healthy VolunteersDown SyndromeAlzheimer s Disease
Perha Pharmaceuticals164 enrolled1 locationNCT06206824
Recruiting
Phase 3

The Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia

AML (Acute Myelogenous Leukemia)Down Syndrome (DS)
Federal Research Institute of Pediatric Hematology, Oncology and Immunology100 enrolled1 locationNCT07232134
Recruiting
Not Applicable

Gamma Frequency Stimulation in Individuals With Down Syndrome

Down Syndrome
Massachusetts Institute of Technology60 enrolled1 locationNCT05196984
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 enrolled215 locationsNCT04546399
Recruiting

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

DementiaAlzheimer DiseaseDown Syndrome
University of Southern California450 enrolled20 locationsNCT04165109
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
Phase 2Phase 3

GnRH Therapy on Cognition in Down Syndrome

Cognitive DeclineAlzheimer Disease, Early OnsetDown Syndrome+1 more
Nelly Pitteloud56 enrolled1 locationNCT04390646
Recruiting
Phase 2

ASIA Down Syndrome Acute Lymphoblastic Leukemia 2016

Acute Lymphoblastic LeukemiaChildhood CancerDown Syndrome
National Hospital Organization Nagoya Medical Center60 enrolled10 locationsNCT03286634