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ClinicalTrials.gov 21 recruiting now early-phase tilt

Down Syndrome: early-phase trial registry

21 of 55 indexed Down Syndrome trials are currently recruiting, with more weight in Phase 1-2.

Corpus placement: #254 of 28,707 conditions by registered trial count.

55 US clinical trials · 21 currently recruiting · #254 of 28,707 conditions by registered trial count

Down Syndrome: 55 US clinical trials tracked, 21 recruiting.

Down Syndrome is the subject of 55 registered US clinical trials on ClinicalTrials.gov, 21 of them currently open to new participants. 5 are in Phase 3-4 (later-stage) and 10 in Phase 1-2 (earlier-stage). The most active sponsor is Sequenom, running 3 of these trials.

Enrollment posture brief

Registry condition down-syndrome

OPEN 21 | RECR 38% | LATE 5 | EARLY 10 | RANK #254 | SPON 3

Nearest open-share peer: Liver Diseases (38% recruiting · 34 trials)

Colorectal Neoplasms41.8%Down Syndrome (this)38%Menopause32.7%Constipation27.3%Brain and Central Nervous Sy1.8%
Open-enrollment share among volume-matched conditions near Down Syndrome

Down Syndrome lists 21 open slots (38% of 55 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →

Key findings

What ClinicalTrials.gov does not surface for Down Syndrome on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.

  1. Down Syndrome recruits in line with its size-band peers.

    21 of 55 indexed trials (38.2%) are open to enrollment, against a 39.7% average across 762 conditions with 20-99 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.

  2. Sponsorship of Down Syndrome trials is spread across many organizations.

    Sequenom accounts for 3 of 55 trials (5.5%). 41 distinct sponsors appear on at least one study for this condition label.

  3. Typical Down Syndrome enrolment sits around 81 participants.

    Median target enrolment is 81 among the 55 trials that report a genuine target (sentinel values excluded). 31 of those (56.4%) plan for fewer than 100 participants.

  4. Down Syndrome research is mostly interventional.

    39 interventional and 16 observational studies make up this index (70.9% interventional). Study type is sponsor-reported on ClinicalTrials.gov.

Phase 12Phase 28Phase 34Phase 41

Open and recent trials

55 total, page 1 of 2

RECRUITING 100,000 participants

DS-Connect®: The Down Syndrome Registry

University of Colorado, Denver

NCT01950624

RECRUITING 2,500 participants

The Human Trisome Project

University of Colorado, Denver

NCT02864108

RECRUITING 2,000 participants

Specimen Collection from Pregnant Women At Increased Risk for Fetal Aneuploidy

Sequenom

NCT01429389

RECRUITING 1,000 participants

Patterns of Neurodevelopmental Disorders

Richard Frye

NCT06213090

RECRUITING 700 participants

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

RECRUITING Phase 2 461 participants

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

RECRUITING 450 participants

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

University of Southern California

NCT04165109

RECRUITING NA 376 participants

Physical Activity and Community EmPOWERment Project

University of North Carolina, Chapel Hill

NCT06740162

RECRUITING 330 participants

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

Children's Oncology Group

NCT05702645

RECRUITING NA 303 participants

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

Oregon Health and Science University

NCT05508971

RECRUITING Phase 2 230 participants

Down Syndrome Obstructive Sleep Apnea

Brigham and Women's Hospital

NCT06043440

RECRUITING Phase 4 100 participants

Evaluating Treatment of ADHD in Children with Down Syndrome

Children's Hospital Medical Center, Cincinnati

NCT04219280

RECRUITING NA 86 participants

PAP for Children With DS and OSAS

University of Miami

NCT04132999

RECRUITING NA 81 participants

Brain Outcomes With Lifestyle Change in Down Syndrome

University of Kansas Medical Center

NCT05985486

RECRUITING NA 60 participants

Measuring Aided Language Development

University of Central Florida

NCT06512168

RECRUITING NA 60 participants

Gamma Frequency Stimulation in Individuals With Down Syndrome

Massachusetts Institute of Technology

NCT05196984

RECRUITING NA 40 participants

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

University of Michigan

NCT05527652

RECRUITING Phase 2 36 participants

Medications for Obstructive Sleep Apnea to Improve Cognition in Children With Down Syndrome

University of Arizona

NCT05933603

RECRUITING NA 30 participants

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

Michigan State University

NCT06951516

RECRUITING NA 20 participants

Sleep Intervention and Quality of Life in Down Syndrome

University of Alabama at Birmingham

NCT06783725

RECRUITING NA 6 participants

Harness-based Mobility Intervention for Infants With Down Syndrome

Boston University Charles River Campus

NCT06219863

ACTIVE NOT RECRUITING Phase 3 9,350 participants

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

ACTIVE NOT RECRUITING Phase 3 6,720 participants

A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia

National Cancer Institute (NCI)

NCT03914625

ACTIVE NOT RECRUITING Phase 3 280 participants

Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome

Children's Oncology Group

NCT02521493

ACTIVE NOT RECRUITING 202 participants

Evaluating Cognitive Outcomes in Down Syndrome

Children's Hospital Medical Center, Cincinnati

NCT04208685

ACTIVE NOT RECRUITING NA 81 participants

Validation of Sleep Questionnaires in the Down Syndrome Population

Oregon Health and Science University

NCT03771469

ACTIVE NOT RECRUITING Phase 2 66 participants

Therapies for Down Syndrome Regression Disorder

University of Colorado, Denver

NCT05662228

ACTIVE NOT RECRUITING NA 60 participants

Proactive Speech and Language Intervention for Infants With Down Syndrome

Arizona State University

NCT06450509

ACTIVE NOT RECRUITING NA 57 participants

Investigating the Impact of JASPER Behavioral Therapy in Children With Down Syndrome

Boston Children's Hospital

NCT05638412

ACTIVE NOT RECRUITING Phase 3 57 participants

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

Inspire Medical Systems

NCT04801771

ACTIVE NOT RECRUITING Phase 2 20 participants

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

Vanderbilt University Medical Center

NCT05231798

ACTIVE NOT RECRUITING NA 19 participants

Transcranial Photobiomodulation With Near-Infrared Light for Language in Individuals With Down Syndrome

Massachusetts General Hospital

NCT04668001

COMPLETED 3,062 participants

Clinical Evaluation of the SEQureDx T21 Test In High Risk Pregnancies

Sequenom

NCT01555346

COMPLETED 2,502 participants

Noninvasive Screening for Fetal Aneuploidy: A New Maternal Plasma Marker

Sequenom

NCT00971334

COMPLETED 760 participants

Expanded Noninvasive Genomic Medical Assessment: The Enigma Study

Progenity

NCT02787486

COMPLETED 273 participants

Congenital Heart Disease: Impact on Learning and Development in Down Syndrome (CHILD-DS)

Carelon Research

NCT05312177

COMPLETED 257 participants

Down Syndrome Metabolic Health Study

Children's Hospital of Philadelphia

NCT01821300

COMPLETED Phase 2 160 participants

Down Syndrome Memantine Follow-up Study

University Hospitals Cleveland Medical Center

NCT02304302

COMPLETED NA 155 participants

A Bicycle Training Intervention

University of Michigan

NCT02046889

COMPLETED Phase 2 109 participants

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

Life Molecular Imaging

NCT00928304

COMPLETED 90 participants

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

COMPLETED NA 44 participants

Strategies for Teaching Verbs

Vanderbilt University Medical Center

NCT03441685

COMPLETED 43 participants

Neurocognitive and Psychosocial Outcomes in Survivors of Childhood Leukemia With Down Syndrome

St. Jude Children's Research Hospital

NCT02859389

COMPLETED NA 42 participants

A Pilot Study to Evaluate the Hypoglossal Nerve Stimulator in Adolescents With Down Syndrome and Obstructive Sleep Apnea

Christopher Hartnick, M.D.

NCT02344108

COMPLETED Phase 1 35 participants

A Study of RG1662 in Individuals With Down Syndrome

Hoffmann-La Roche

NCT01436955

COMPLETED NA 28 participants

Oxygen Uptake Kinetics During Submaximal Exercise in Adults With Down Syndrome

University of Nevada, Las Vegas

NCT05995223

COMPLETED Phase 2 22 participants

Medications for Obstructive Sleep Apnea In Children With Down Syndrome

University of Arizona

NCT04115878

COMPLETED Phase 1 20 participants

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

AC Immune

NCT02738450

COMPLETED NA 19 participants

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

St. Joseph's Hospital and Medical Center, Phoenix

NCT02759887

COMPLETED NA 18 participants

Investigating Trunk Control in Young Children With Down Syndrome

Texas Woman's University

NCT03544840

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Phase Distribution

PhaseTrial count
Phase 1 2
Phase 2 8
Phase 3 4
Phase 4 1

Named interventions

Most-linked intervention names on Down Syndrome records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.

Cytarabine Drug
3 trials
Placebo Drug
3 trials
Asparaginase Erwinia chrysanthemi Drug
2 trials
Blinatumomab Biological
2 trials
Dexamethasone Drug
2 trials
Biospecimen Collection Procedure
2 trials
Cyclophosphamide Drug
2 trials
Blood sampling for Laboratory Developed Test (LDT) analysis Other
1 trials

US sites by state

Facility locations filed on Down Syndrome trials. A trial with sites in several states counts in each.

Arizona 17 trials
California 15 trials
Ohio 11 trials
Massachusetts 10 trials
Pennsylvania 9 trials
Michigan 9 trials
Alabama 9 trials
Colorado 7 trials

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

What to do with this Down Syndrome page

55 registered trials, 21 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.

  • 21 Down Syndrome trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
  • Sequenom sponsors the most Down Syndrome trials on record, review their full trial history before evaluating a specific study. See Sequenom's trials
  • Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide

Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Down Syndrome condition card totals 55 registered trials; most-linked intervention Cytarabine; top US site state Arizona. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.