19 completed, 10 recruiting, 3 withdrawn, 2 active not recruiting. 39 Phase 2, 4 Phase 1, 2 Phase 3.
Follow Down Syndrome: new trials by email Literature Review of Down Syndrome trials Research Report on Down Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| An Open-Label Phase II Feasibility Study for the Use of Ublituximab in Adults With Down Syndrome Regression Disorder Ublituximab - Down Syndrome Regression Disorder (DSRD), Down Syndrome (Trisomy 21) | Phase 2 | Not yet recruiting | Jonathan Santoro | 20 | 2026-10-01 | 2026-09-23 |
| A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD) Mivelsiran, Placebo - Down Syndrome-Associated Alzheimer's Disease (DS-AD) | Phase 2 | Recruiting | Alnylam Pharmaceuticals | 58 | 2026-08-17 | 2026-10-13 |
| Metric-Optimized Spectacle Prescriptions for Children With Down Syndrome Metric-optimized spectacles, Routine Clinical Spectacles - Refractive Error Correction, Visual Acuity | Phase 2 | Recruiting | Ohio State University | 24 | 2026-07-13 | 2026-07-20 |
| Modulation of the Immune System in Down Syndrome for Improved Outcomes and Neurodevelopment - 1 Tofacitinib Oral Solution, Placebo - Down Syndrome | Phase 2 | Recruiting | University of Colorado, Denver | 92 | 2026-05-28 | 2026-09-03 |
| Treatment of Atopic Dermatitis and Alopecia Areata With Abrocitinib in Individuals With Down Syndrome Abrocitinib - Atopic Dermatitis, Alopecia Areata | Phase 2 | Recruiting | Icahn School of Medicine at Mount Sinai | 56 | 2026-01-12 | 2026-10-16 |
| AEF0217 in Participants With Down Syndrome AEF0217 100 µg, AEF0217 300 µg, Placebo - Down Syndrome (Trisomy 21) | Phase 2 | Recruiting | Aelis Farma | 188 | 2025-12-22 | 2026-04-08 |
| Down Syndrome Obstructive Sleep Apnea Oxygen - Down Syndrome, Obstructive Sleep Apnea | Phase 2 | Recruiting | Brigham and Women's Hospital | 230 | 2023-10-24 | 2026-09-30 |
| Trial to Evaluate Safety and Efficacy of GM-CSF /Sargramostim in Down Syndrome Sargramostim for Injection, Saline Placebo - Down Syndrome | Phase 2 | Withdrawn | University of Colorado, Denver | 2023-10-23 | 2025-04-02 | |
| Medications for Obstructive Sleep Apnea to Improve Cognition in Children With Down Syndrome Atomoxetine and Oxybutynin (ato-oxy) - Obstructive Sleep Apnea, Down Syndrome | Phase 2 | Active not recruiting | University of Arizona | 36 | 2023-09-29 | 2026-04-07 |
| Therapies for Down Syndrome Regression Disorder Lorazepam, Intravenous immunoglobulin (IVIG), Tofacitinib - Down Syndrome, Regression | Phase 2 | Completed | University of Colorado, Denver | 66 | 2023-06-29 | 2026-09-03 |
| Efficacy of Bumetanide to Improve Cognitive Functions in Down Syndrome Bumetanide, Placebo - Down Syndrome | Phase 2 | Unknown | Stefano Vicari | 64 | 2023-01-11 | 2024-06-20 |
| Phase 1/2 Trial of AEF0217 in Participants With Down Syndrome Placebo, AEF0217 - Down Syndrome | Phase 1 / Phase 2 | Completed | Aelis Farma | 40 | 2022-12-15 | 2024-10-10 |
| A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study) Placebo (Study Part 1a), ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a - Amyloid Plaque, Beta-Amyloid | Phase 1 / Phase 2 | Recruiting | AC Immune SA | 304 | 2022-06-21 | 2026-07-10 |
| Dilated Versus Non-Dilated Wavefront Corrections for Patients With Down Syndrome Dilated Refraction, Non-Dilated Refraction - Down Syndrome, Refractive Errors | Phase 2 | Completed | Ohio State University | 42 | 2022-05-06 | 2026-05-08 |
| A Study of ACI-24 in Adults With Down Syndrome ACI-24, Placebo - Down Syndrome | Phase 2 | Withdrawn | AC Immune SA | 2021-10-01 | 2021-10-22 | |
| Cholinergic Integrity in Down Syndrome in Association With Aging, Alzheimer's Disease Pathology, and Cognition [18F]-FEOBV Radiotracer - Down Syndrome, Down Syndrome, Partial Trisomy 21 | Phase 2 | Recruiting | Vanderbilt University Medical Center | 30 | 2021-08-19 | 2026-06-22 |
| A Study to Compare Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL) 3-Dimensional Conformal Radiation Therapy, Asparaginase Erwinia chrysanthemi, Biospecimen Collection - Down Syndrome, Recurrent B Acute Lymphoblastic Leukemia | Phase 2 | Recruiting | National Cancer Institute (NCI) | 461 | 2020-12-17 | 2026-09-01 |
| Medications for Obstructive Sleep Apnea In Children With Down Syndrome Atomoxetine and oxybutynin (ato-oxy) - Obstructive Sleep Apnea, Down Syndrome | Phase 2 | Completed | University of Arizona | 22 | 2020-10-21 | 2025-08-19 |
| Tofacitinib for Immune Skin Conditions in Down Syndrome Tofacitinib - Down Syndrome, Alopecia Areata | Phase 2 | Completed | University of Colorado, Denver | 47 | 2020-10-21 | 2025-12-11 |
| GnRH Therapy on Cognition in Down Syndrome GnRH, gonadorelin acetate, 0.9% NaCl - Down Syndrome, Cognitive Decline | Phase 2 / Phase 3 | Recruiting | Nelly Pitteloud | 56 | 2020-08-27 | 2024-02-12 |
Showing 20 of 39. Sign in to see all of them.
Alzheimer Disease (5) · Cognitive Dysfunction (4) · Sleep Apnea, Obstructive (3) · Alopecia Areata (2) · Autoimmune Diseases (2) · Dementia (2) · Dermatitis, Atopic (2) · Plaque, Amyloid (2) · Autoimmune Diseases of the Nervous System (1) · Behavior (1) · Brain Diseases (1) · Burkitt Lymphoma (1) · Catatonia (1) · Central Nervous System Diseases (1)
Abnormalities, Multiple (39) · Chromosome Disorders (39) · Congenital Abnormalities (39) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (39) · Genetic Diseases, Inborn (39) · Nervous System Diseases (39) · Neurobehavioral Manifestations (39) · Neurologic Manifestations (39) · Intellectual Disability (38) · Mental Disorders (9) · Neurocognitive Disorders (7) · Pathological Conditions, Signs and Symptoms (7) · Immune System Diseases (5) · Neurodegenerative Diseases (5) · Tauopathies (5)
tofacitinib (3) · Atomoxetine Hydrochloride (2) · Cytarabine (2) · Donepezil (2) · Hydrocortisone (2) · Mercaptopurine (2) · Methotrexate (2) · Vincristine (2) · epigallocatechin gallate (2) · oxybutynin (2) · 4-(N-methylamino)-4'-(2-(2-(2-fluoroethoxy)ethoxy)ethoxy)stilbene (1) · Asparaginase (1) · Biopsy (1) · Bumetanide (1) · Calcium Dobesilate (1)
University of Colorado, Denver (4) · AC Immune SA (2) · Aelis Farma (2) · Eisai Inc. (2) · Hoffmann-La Roche (2) · Ohio State University (2) · Parc de Salut Mar (2) · University of Arizona (2) · Vanderbilt University Medical Center (2) · Alnylam Pharmaceuticals (1) · Asklepieion Voulas General Hospital (1) · Brigham and Women's Hospital (1) · Children's Hospital Medical Center, Cincinnati (1) · Duke University (1) · Eisai Co., Ltd. (1)
United States (26) · Spain (6) · France (3) · Italy (3) · Canada (2) · Japan (2) · Mexico (2) · Singapore (2) · United Kingdom (2) · Argentina (1) · Australia (1) · Brazil (1) · Egypt (1) · Greece (1) · Hong Kong (1)
Joaquin Espinosa, PhD (3) · Alberto C Costa, MD, PhD (1) · Alexander C Conley, Ph.D. (1) · Alexander C Conley, PhD (1) · Allen Yeoh, MBBS (1) · Clotilde Mircher, MD (1) · Daniel Combs, MD (1) · Dusan Bogunovic, PhD (1) · Elise Sannar, MD (1) · Emma Guttman, MD (1) · Emmanouil Gkliatis, MD (1) · Francis Hickey, M.D. (1) · George Capone, MD (1)
PMID 20084109, PMID 27302362, PMID 28453471, PMID 33773598, PMID 33778150, PMID 34535086, PMID 34942135, PMID 35123401, PMID 35307513, PMID 35622074, PMID 36048943, PMID 37555595, PMID 38946973, PMID 39737640, PMID 41671463
2025 (2), R01DC016321 (1), R01EY024590 (1), R33AR077495 (1), R33HL151254 (1), R61AG074859 (1), R61AR084210 (1), R61HL151254 (1), U10CA180886 (1)