11 completed, 2 not yet recruiting, 1 enrolling by invitation, 1 recruiting. 16 Phase 1, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 16 | Completed 11, Not yet recruiting 2, Enrolling by invitation 1, Recruiting 1, Terminated 1 |
| Phase 2 | 4 | Completed 3, Recruiting 1 |
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 |
|---|---|---|---|---|---|---|
| Escalating Doses of Memantine in Down Syndrome (MEDS-123) Memantine - Down Syndrome, Intellectual Disability | Phase 1 | Not yet recruiting | University Hospitals Cleveland Medical Center | 25 | 2026-10-14 | 2026-10-05 |
| NAD Augmentation to Prevent or Reverse Alzheimer's Disease in People With Down Syndrome MIB-626, Placebo - Down Syndrome (DS) | Phase 1 | Not yet recruiting | Brigham and Women's Hospital | 24 | 2026-08-31 | 2026-08-19 |
| Hero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) and Pharmacodynamics (PD) of ION269 in Participants With Down Syndrome (DS) at Risk for Alzheimer's Disease (AD) ION269 - Alzheimer Disease, Down Syndrome | Phase 1 | Terminated | Ionis Pharmaceuticals, Inc. | 1 | 2024-12-20 | 2026-02-20 |
| Leucettinib-21 First-in-Human Phase 1 in Healthy Volunteers and Subjects With Down Syndrome and Alzheimer's Disease Leucettinib-21 - Healthy Volunteers, Down Syndrome | Phase 1 | Completed | Perha Pharmaceuticals | 152 | 2024-01-18 | 2026-06-30 |
| Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities Methylphenidate Oral Solution - Intellectual Disability, Fragile X Syndrome | Phase 1 | Enrolling by invitation | University of California, Davis | 68 | 2023-02-01 | 2025-12-15 |
| 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 |
| Safety, Tolerability and Immunogenicity of ACI-24 Vaccine in Adults With Down Syndrome ACI-24 low dose, ACI-24 high dose, Placebo - Down Syndrome | Phase 1 | Completed | AC Immune SA | 20 | 2016-03-01 | 2021-10-15 |
| Investigation of the Safety of Intranasal Glulisine in Down Syndrome Insulin glulisine, Saline - Down Syndrome | Phase 1 | Completed | HealthPartners Institute | 12 | 2015-04-01 | 2019-12-06 |
| Parents Resources for Decreasing the Incidence of Change Triggered Temper Outbursts Visual Scheduling, signalling change - Child Behavior | Phase 1 | Completed | Queen's University, Belfast | 80 | 2015-01-01 | 2020-02-17 |
| Nicotine Treatment of Cognitive Decline in Down Syndrome Low Dose Nicotine (7mg), Moderate Dose Nicotine (14mg) - Down Syndrome, Mild Cognitive Impairment | Phase 1 / Phase 2 | Completed | Vanderbilt University Medical Center | 7 | 2013-04-01 | 2022-05-10 |
| A Molecular and Functional Brain Imaging Study in Individuals With Down Syndrome and Healthy Controls Following Single Dose RG1662 RG1662, placebo - Healthy Volunteer, Down Syndrome | Phase 1 | Completed | Hoffmann-La Roche | 13 | 2012-08-01 | 2015-09-02 |
| A Study of RG1662 in Individuals With Down Syndrome Placebo, RG1662 - Down Syndrome | Phase 1 | Completed | Hoffmann-La Roche | 35 | 2011-11-01 | 2015-03-03 |
| An Interactive Informed Consent and Education Program for Pregnant Women Information about Prenatal Screening and Diagnosis - Pregnancy, Down Syndrome | Phase 1 | Completed | Northwestern University | 150 | 2010-08-01 | 2014-11-07 |
| Rivastigmine Study in Adolescents With Down Syndrome Rivastigmine, Liquid Placebo - Down Syndrome | Phase 1 / Phase 2 | Completed | Duke University | 42 | 2009-11-01 | 2015-04-06 |
| Rehabilitation of Children With Multiple Disabilities CTN model of care for children and their families - Cerebral Palsy, Autism | Phase 1 | Completed | Children's Treatment Network | 445 | 2007-04-01 | 2011-06-23 |
Alzheimer Disease (5) · Intellectual Disability (3) · Autistic Disorder (2) · Cognitive Dysfunction (2) · Dementia (2) · Fragile X Syndrome (2) · Attention Deficit Disorder with Hyperactivity (1) · Brain Diseases (1) · Brain Injuries (1) · Central Nervous System Diseases (1) · Cerebral Palsy (1) · Child Behavior (1) · Plaque, Amyloid (1) · Prader-Willi Syndrome (1)
Abnormalities, Multiple (16) · Chromosome Disorders (16) · Congenital Abnormalities (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Genetic Diseases, Inborn (16) · Nervous System Diseases (16) · Neurobehavioral Manifestations (16) · Neurologic Manifestations (16) · Intellectual Disability (13) · Mental Disorders (9) · Neurocognitive Disorders (6) · Brain Diseases (5) · Central Nervous System Diseases (5) · Neurodegenerative Diseases (5) · Tauopathies (5)
Diagnosis (1) · Insulin (1) · Memantine (1) · Methylphenidate (1) · Nicotine (1) · Rivastigmine (1) · Sodium Chloride (1) · Tobacco Use Cessation Devices (1) · insulin glulisine (1)
AC Immune SA (2) · Hoffmann-La Roche (2) · Aelis Farma (1) · Brigham and Women's Hospital (1) · Children's Treatment Network (1) · Duke University (1) · HealthPartners Institute (1) · Ionis Pharmaceuticals, Inc. (1) · Northwestern University (1) · Perha Pharmaceuticals (1) · Queen's University, Belfast (1) · University Hospitals Cleveland Medical Center (1) · University of California, Davis (1) · Vanderbilt University Medical Center (1)
United States (11) · United Kingdom (4) · Spain (3) · Canada (1) · France (1)
Alberto C Costa, MD, PhD (1) · Alexander C Conley, PhD (1) · Bill Grobman, M.D., M.B.A (1) · George Capone, MD (1) · Kate Woodcock, PhD (1) · Michael H Rosenbloom, MD (1) · Michael Rafii, MD (1) · Michael S. Rafii, MD, PhD (1) · Michael Wolf, Ph.D, M.P.H (1) · Paul A Newhouse, MD (1) · Priya Kishnani, MD (1) · Robert Levine, M.D. (1) · Sandy Thurston (1)
2023 (2), R01AG047922 (1), R01HD076189 (1)