Clinical Trials for Down Syndrome

Source: ClinicalTrials.gov — U.S. National Library of Medicine — A database of privately and publicly funded clinical studies conducted around the world (U.S. National Library of Medicine, public domain).

This page lists clinical trials related to Down Syndrome from the ClinicalTrials.gov database.

Trial Summary

• Total studies: 50
• Recruiting: 22
• Active, not recruiting: 5
• Completed: 14
• Other: 9

Results are refreshed periodically and may not reflect the most current information available from ClinicalTrials.gov.

Recruiting Trials

NCT04546399 — A Study to Compare Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL)

This phase II trial studies the effect of nivolumab in combination with blinatumomab compared to blinatumomab alone in treating patients with B-cell acute lymphoblastic leukemia (B-ALL) that has come back (relapsed). Down syndrome patients with relapsed B-ALL are included in this study. Blinatumomab is an antibody, which is a protein that identifies and targets specific molecules in the body. B…

NCT04165109 — Trial-Ready Cohort-Down Syndrome (TRC-DS)

The purpose of the Trial-Ready Cohort - Down Syndrome (TRC-DS) is to enroll 120 healthy adults with Down syndrome (DS), between the ages of 25-55, into a trial ready cohort (TRC), and up to 550 participants in total including co-enrolled in the Alzheimer Biomarkers Consortium - Down Syndrome (ABC-DS) study. Participants enrolled in the TRC-DS will undergo longitudinal cognitive and clinical ass…

NCT05231798 — Cholinergic Integrity in Down Syndrome in Association With Aging, Alzheimer’s Disease Pathology, and Cognition

Progressive age-related cognitive deficits occurring in both AD and DS have been connected to the degeneration of several neuronal populations, but mechanisms are not fully elucidated. The most consistent neuronal losses throughout the progression of AD are seen in cholinergic neurons where these losses negatively affect cognition, particularly in attention, learning, and memory formation. Evid…

NCT04726241 — 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

This study aims to use clinical and biological characteristics of acute leukemias to screen for patient eligibility for available pediatric leukemia sub-trials. Testing bone marrow and blood from patients with leukemia that has come back after treatment or is difficult to treat may provide information about the patient’s leukemia that is important when deciding how to best treat it, and may hel…

NCT05462106 — A Study to Assess the Effects of ACI-24.060 in Alzheimer’s Disease and in Down Syndrome (ABATE Study)

The purpose of this study is to assess the safety, tolerability, immunogenicity and pharmacodynamic effects of ACI-24.060 in subjects with prodromal Alzheimer’s disease and in non-demented adults with Down syndrome.

NCT05508971 — Treatment of Obstructive Sleep Apnea With Personalized Surgery in Children With Down Syndrome (TOPS-DS)

The overall objective of this randomized clinical trial is to test the effectiveness of a personalized approach to the surgical treatment of OSA in children with Down syndrome (DS).The estimated prevalence of obstructive sleep apnea (OSA) in children with DS ranges from 45-83%, compared to 1-6% in the general pediatric population. Untreated OSA in children has been associated with daytime sleep…

NCT05702645 — A Study to Learn More About the Health of Persons With Down Syndrome After Treatment for Acute Leukemia

This study attempts to learn more about the health of persons with Down syndrome after treatment for acute leukemia. Children with Down syndrome are at increased risk for side effects during treatment for acute leukemia, but it is unclear of their risk for long-term effects of cancer treatment. By learning more about the factors that may contribute to chronic health conditions and long-term eff…

NCT06213090 — Patterns of Neurodevelopmental Disorders

The purpose of this study is to systematically evaluate the results of medical investigations to identify symptom and biological patterns and common etiologies of neurodevelopmental disorders.

NCT06951516 — How Simplified Language Affects Comprehension and Learning in Young Children With Down Syndrome

The long-term study goal is to experimentally evaluate the components (and likely active ingredients) of early language interventions for young children with Down syndrome (DS). The overall objective is to determine how single-word and telegraphic simplification affects real-time language processing and word learning in young children with DS (relative to full, grammatical utterances). The prop…

NCT06866925 — tDCS for Catatonic Depression in Down Syndrome: A Pilot Study

This study evaluates the efficacy of transcranial direct current stimulation (tDCS) for depression with catatonia in individuals with Down syndrome (DS). 62 patients will be randomized to receive 15 sessions of active or sham tDCS. The primary objective is to measure changes in depressive/catatonic symptoms using the Bush-Francis Catatonia Rating Scale (BFCRS). Secondary objectives include safe…

NCT07578922 — Effects of Bean Bag Tossing Game on Balance and Gait in Children With Down Syndrome.

The research design will be a randomized clinical trial. The study will recruit 36 children with spastic cerebral palsy and toe walking that fall within the ages of 5-17 years who have a defined balance deficit. The participants will be randomly assigned to one of two groups: group A (n=18), which will play the Bean Bag Tossing Game on Wedge, and group B (n=18), which will play the Bean Bag Tos…

NCT07643896 — 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

The goal of the ADVANCE (Assay Development and Validation for Pre-Natal and Obstetric Conditions) study is to compare the concordance of results of a novel non-invasive circulating fetal cell (CFC) assay to the results of prenatal invasive diagnostic testing or postnatal genetic and clinical diagnosis of the resulting neonate. This is a prospective study of pregnant individuals.

NCT07156318 — Drumming Lessons’ Influence on Children With Down Syndrome

The goal of this clinical trial is to learn if drumming lessons can increase self-control in children with Down syndrome. The main question it aims to answer is whether 2 months of drumming lessons can improve the behavioral control and timing skills in children with Down syndrome. Participants are between 7 and 15 years of age and receive two months of drumming lessons given by a professional …

NCT07425769 — Adapting RDAD for DS

The goal of this clinical trial is to observe feasibility and initial efficacy of a remotely delivered exercise and dementia caregiving intervention in older adults with Down syndrome and their caregivers.

NCT07579559 — Animal Fun Program and Kinesthetic Development in Down Syndrome

The purpose of the study is that Animal-fun activities capture children’s attention, making them a powerful tool for enhancing kinesthetic awareness and gross motor skills. Although play-based programs like Animal Fun boost motor development, no research has yet examined if adding animal interaction can specifically improve proprioception in children with Down syndrome. There is no difference i…

NCT07238465 — Exploring Sympathetic Nervous System Function in Individuals With Down Syndrome

Down syndrome (DS), the most common genetic cause of intellectual disability, is associated with widespread organ dysfunction, including abnormalities in the autonomic nervous system (ANS). The ANS regulates critical functions such as heart rate (HR) and blood pressure (BP), both essential for maintaining homeostasis and supporting physical activity. Individuals with DS often exhibit blunted HR…

NCT07630207 — Vascular Function in Adults With Down Syndrome

Adults with Down syndrome (Ds) are often thought to have a lower risk of heart and blood vessel disease because they tend to have lower blood pressure and fewer heart attacks than people without Ds. However, recent research suggests that heart and blood vessel diseases, including stroke, are becoming a more common cause of death in adults with Ds as life expectancy increases. Despite these find…

NCT07296861 — HomeGrown: A Family-based Lifestyle Intervention to Support Healthy Development of Young Children With Down Syndrome

The goal of this project is to evaluate an adapted health promotion program, HomeGrown, designed to improve the health of young children with Down syndrome by supporting families in making healthy home environmental changes. There is a significant need for evidence-based programs that address healthy eating and physical activity within this population, as most existing interventions have been d…

NCT07598643 — Modulation of the Immune System in Down Syndrome for Improved Outcomes and Neurodevelopment - 1

This protocol describes a phase 2, double-blind, randomized, placebo-controlled clinical trial for Janus kinase (JAK) inhibition in Down syndrome (DS). This trial will evaluate the safety and efficacy of a 6-month treatment with the JAK1/3 inhibitor tofacitinib (XELJANZ) in individuals ages 6-22 (inclusive) with DS. There will be two main arms for this study: a treatment arm and a placebo contr…

NCT07625579 — Collaboration for Down Syndrome Progress (CDP)

The Collaboration for Down Syndrome Progress (CDP) is a long-term study that follows people with Down syndrome of all ages. The goal is to better understand their health, development, and everyday experiences over time. Participants and their caregivers will answer questions, share medical information, and may give samples like blood or saliva. Some participants may also take part in optional a…

NCT07490444 — Metric-Optimized Spectacle Prescriptions for Children With Down Syndrome

This study seeks to determine whether glasses prescriptions determined from computer analysis of detailed images of the eyes provide better vision outcomes for children with Down syndrome than prescriptions determined using routine clinical methods.

NCT07416201 — Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy

Background:

Down syndrome is a genetic disorder that can cause heart defects and other problems in the body. People with Down syndrome are more likely to have infections, autoimmunity, and blood diseases. Some may need surgery to treat congenital heart problems. During this surgery, doctors sometimes remove part of the thymus. The thymus is an organ that plays a role in immune function. People…

Active, Not Recruiting

NCT03286634 — ASIA Down Syndrome Acute Lymphoblastic Leukemia 2016

To evaluate the outcome of a prednisolone and low dose methotrexate based protocol in Down syndrome children with ALL (DS-ALL) in an Asia-wide study. The treatment protocol was modified based upon backbone of Taiwan Pediatric Oncology Group (TPOG)-ALL protocol in which risk classification will be guided by level of flow minimal residual disease (MRD) instead.

NCT03914625 — A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia

This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients with newly diagnosed, standard risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down syndrome. Monoclonal antibodies, such as blinatumomab, may induce changes in the body’s immune system and may interfere with the ability of cancer cells to grow and spread. Che…

NCT05146739 — Highest Dose of Uproleselan in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Mixed Phenotype Acute Leukemia Relapsed or Refractory That Expresses E-selectin Ligand on the Cell Membrane

This phase I trial tests the safety, side effects, and determination of the best dose of uproleselan in combination with fludarabine and cytarabine in treating patients with acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that has come back (relapsed) or does not respond to treatment (refractory) and that expresses E-selectin ligand on the cell membrane. Uprol…

NCT06465485 — STEP: Phase IIIb Study of Benralizumab to Step-down Maintenance Therapy in Patients With Severe Eosinophilic Asthma

This is a prospective, multi-center, single-arm Phase 3b study designed to evaluate the potential benefit to patients if benralizumab treatment could enable reduction in asthma maintenance controllers while allowing patients to maintain asthma control in Chinese patients.

NCT07328165 — The Impact of Core Stability Versus Respiratory Muscle Strengthening Exercises on Bone Mineral Density in Children With Down Syndrome

Children with Down syndrome often exhibit diminished physical fitness levels and lower bone mass. Numerous factors contribute to this decrease in bone mass density, including decreased levels of leptin, reduced muscle mass and strength, impaired balance, and skeletal abnormalities. These factors further exacerbate the risk of developing osteoporosis in this population. Therefore, it is crucial …

Not Yet Recruiting

NCT07531940 — Escalating Doses of Memantine in Down Syndrome (MEDS-123)

Down syndrome (DS) is typically caused by an extra chromosome 21 in the cell nucleus (trisomy 21, or T21). T21 is both the most common cause of genetically defined intellectual disability and the earliest documented cause of Alzheimer’s disease (AD)-type pathology. Currently, all presymptomatic individuals with DS are classified as having ‘Stage 0’ DS-associated AD (DSAD). DSAD pathology evolve…

NCT07658053 — Effects of a Pacifier on Obstructive Sleep Apnea and Its Repercussions in Infants With Down Syndrome

Obstructive Sleep Apnea (OSA) is characterised by repetitive collapse of the upper airway during sleep, inducing breathing disturbances that can result in oxygen desaturation and frequent arousals. In children, OSA can have long-term consequences on the development and on the cardiovascular system.

Down Syndrome (DS) is a genetic disorder associated with intellectual disability and many comorb…

NCT07636811 — A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer’s Disease (DS-AD)

The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month open-label treatment extension (OLE) period. The estimated duration of study participation, inclusiv…

NCT07278492 — NAD Augmentation to Prevent or Reverse Alzheimer’s Disease in People With Down Syndrome

The aim of the study is to determine the safety and tolerability of different increasing doses of MIB-626 (a microcrystalline form of β nicotinamide mononucleotide; NMN) given daily for 28 consecutive days to adults with Down syndrome (DS). The study will also explore how the drug moves through the body over time (pharmacokinetics or PK) and how the drug affects the body in different ways (phar…

NCT06911944 — Amyloid Lowering for Alzheimer’s in Down’s With Donanemab Investigation

The goal of this clinical trial is to learn if donanemab can reduce levels of amyloid in the brain, and if donanemab is safe and well-tolerated in participants with Down syndrome.

The main questions it aims to answer are: Does donanemab reduce amyloid in the brain? Is donanemab safe and well-tolerated in people with Down syndrome? Researchers will compare donanemab to a placebo (a look-alike s…

NCT07577791 — Prone Row Exercise Versus Lateral Pull Down Exercise In Shoulder Impingement Syndrome With Scapular Dyskinesia

The purpose of this study is to compare the prone row exercise, and the lateral pull down exercise, in individuals diagnosed with shoulder impingement syndrome and scapular dyskinesia regarding latissimus dorsi strength, middle trapezius strength, lower trapezius strength, rhomboids strength, Visual analog scale score, Shoulder pain and disability index score and scapular stability test values.

NCT07631130 — Home and Community Use of a Suspension Walker in Pre-Walking Infants With Down Syndrome

The goal of this clinical trial is to learn if pre-walking infants with Down syndrome can use a suspension walker in their home and community environments. The main questions it aims to answer are:

• Is suspension walker intervention feasible for pre-walking infants with Down syndrome?
• What are barriers to successfully using suspension walkers in home and community environments?
• What are f…

Enrolling by Invitation

NCT07668908 — Immersive Virtual Reality Program in Down Syndrome

The presented project aims to bring immersive technology to individuals with Down syndrome (DS) through the development of innovative proposals for acquiring skills and abilities, and enhancing their capabilities by combining the motor and cognitive components so important for this group. Various strategies have been used to improve the functionality (physical and cognitive) and quality of life…

Completed Trials

NCT01190930 — Risk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic Lymphoma

This partially randomized phase III trial studies the side effects of different combinations of risk-adapted chemotherapy regimens and how well they work in treating younger patients with newly diagnosed standard-risk acute lymphoblastic leukemia or B-lineage lymphoblastic lymphoma that is found only in the tissue or organ where it began (localized). Drugs used in chemotherapy work in different…

NCT07653399 — Neurodevelopmental Treatment for Balance and Mobility in Children With Down Syndrome

The goal of this clinical trial is to evaluate whether Neurodevelopmental Treatment (NDT) can improve balance, mobility, walking capacity, and functional independence in preschool children with Down syndrome.

The main questions it aims to answer are:

• Does an 8-week Neurodevelopmental Treatment program improve balance performance in children with Down syndrome?
• Does Neurodevelopmental Trea…

NCT04631237 — Developing a Down Syndrome Health Instrument

Although over 200,000 individuals with DS live in the United States, studies to date have focused on outcomes apart from health. The foundation for this proposal is based on the need to accurately measure health of all individuals - specifically, with DS - and the dearth of available tools for this population. Creating such an instrument will provide a barometer of the current state of health f…

NCT04132999 — Positive Airway Pressure (PAP) for Children With Down Syndrome (DS) and Obstructive Sleep Apnea Syndrome (OSAS)

Determine the efficacy of family-informed intervention (INT) vs standard clinical care over a period of twelve months in children with obstructive sleep apnea and Down Syndrome.

NCT05059041 — Dilated Versus Non-Dilated Wavefront Corrections for Patients With Down Syndrome

Individuals with Down syndrome (DS) live with visual deficits due, in part, to elevated levels of higher-order optical aberrations (HOA). HOAs are distortions/abnormalities in the structure of the refractive components of the eye (i.e. the cornea and the lens) that, if present, can result in poor quality focus on the retina, thus negatively impacting vision. HOAs in the general population are o…

NCT06042049 — A Study to Assess Safety, Pharmacokinetics Anti-Drug Antibody and Anti-RSV Antibody After 2 Doses of Nirsevimab

The purpose of this study is to measure the safety, PK, occurrence of ADA to nirsevimab, and anti-RSV neutralizing Ab in Japanese children with certain health conditions or pre-term infants aged ≤12 months.

Study details include

• The study duration is approximately 21 months with a 2-month enrollment period.
• Study intervention is 2 doses administered 5- 6 months apart.
• The study has 5 or…

NCT06592404 — The (Cost)Effectiveness of a Social Robot for Persons With Problems in Daily Structure and Planning in Disability Care

The goal of this multiple baseline single case study is to study the (cost)effectiveness of a social robot in reducing professional caregiver support and promoting independence for individuals in long-term disability care experiencing problems with daily structure and planning. The main research questions it aims to answer are:

• What is the effect of the social robot on the frequency of momen…

NCT06206824 — Leucettinib-21 First-in-Human Phase 1 in Healthy Volunteers and Subjects With Down Syndrome and Alzheimer’s Disease

Leucettinib-21 First-in-Human Phase 1 Study in 6 Parts: Single (Part 1 and 5) and Multiple (Part 3 and 6) Ascending Doses, and Food-Effect (Part 2) in Healthy Subjects, and Single Dose (Part 4) in People with Down Syndrome (DS) and Alzheimer’s Disease (AD).

For Parts 1, 3, 4, 5 and 6, safety and tolerability of an oral administration of Leucettinib-21 will be assessed as primary objectives. Ph…

NCT06450509 — Proactive Speech and Language Intervention for Infants With Down Syndrome

Children with Down syndrome (DS) face life-long struggles with verbal communication. Babble and speech sound development is delayed, and speech can be difficult to understand. Words emerge late, at 21 months on average, compared to 12 months for typical peers, and vocabulary and grammar can remain limited throughout adulthood. Because DS is diagnosed at or even before birth, these difficulties …

NCT06036641 — The Effects of Prolonged Head-Down Tilt Lithotomy Position on Lower Limb Haemodynamics

During certain bowel surgeries for cancer (colorectal surgery), individuals frequently have to be placed in a head-down position to enable surgeons have optimal access to the cancer site. This position usually also involves bending the hips and knees while supporting the legs in stirrups. However, lying in this position for long periods of time can lower the flow of blood to the legs, which can…

NCT06617637 — Effects of Aerobic-Based Virtual Reality Exercise Training and Traditional Aerobic Exercise Training on Physical Fitness, Functional Capacity and Cognitive Function in Individuals With Down Syndrome

This study aims to investigate the effectiveness of aerobic-based virtual reality exercise training and traditional aerobic exercise training on physical fitness, functional capacity, cognitive functions and quality of life in individuals with Down syndrome.

NCT06591559 — Up, Down, and All Around: Evaluating Mobility Devices for Young Children With Developmental Disabilities and Delays

This research study will examine young children with Down syndrome’s initial experiences with mobility devices. Children’s biomechanics and exploration will be quantified while they are using both an overground partial bodyweight support system and powered mobility device.

NCT07534085 — Effects of a Structured Physical Activity Program on Balance and Functional Performance in Children With Down Syndrome

Children with Down syndrome commonly exhibit hypotonia, impaired balance, and reduced functional mobility. This study aimed to evaluate the effects of a structured yoga-based physical activity program on balance and functional performance in children with Down syndrome.

Fifteen children aged 7-12 years participated in a 12-week intervention program consisting of adaptive yoga exercises, breath…

NCT07182071 — 10-Week Physical Activity Program for Mothers of Children With Developmental Disabilities

The purpose of this study is to determine the feasibility and acceptability of a 10-week supervised Physical Activity program for mothers of children with developmental disabilities. 30 participants will be enrolled: 15 into an Intervention group and 15 into a Waitlist Control group.

Other (Terminated)

NCT06042257 — Guanfacine for Hyperactivity in Children With Down Syndrome (HYPEbeGONE_DS)

The primary purpose of this study is to determine efficacy of guanfacine immediate release (GIR) for the treatment of hyperactivity/impulsivity and inattention in children 6-12 years of age with Down syndrome (DS) after 8 weeks of treatment.