Clinical trials
Down Syndrome clinical trials
Below are recruiting down syndrome clinical trials, each written for real people, not researchers. We’re tracking 3,048 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05361811Recruiting
Therapy coaching for parents of children with rare genetic syndromes
This trial tests an “Acceptance and Commitment Therapy” program for parents or legal guardians who feel moderate parenting stress. It aims to help caregivers cope in healthier ways and may also improve how they manage stress while supporting their child.
Bethesda, MarylandAges 18 years+ - NCT05292664RecruitingPhase 1
Venetoclax trial for high-risk blood cancers in young patients
This Phase 1 study tests venetoclax to treat certain high-risk blood cancers in children, teens, or young adults. It is designed to see if the drug can work safely for patients whose disease has returned or did not respond to prior treatment.
San Francisco, CaliforniaAges 1–40 - NCT05085704Recruiting
Brain scans to study metabolism in healthy and diabetic people
This study uses MRI brain scans at different strengths to measure how the brain uses energy (metabolism) in health and in people with genetically confirmed G1D. It may help researchers understand brain energy changes related to metabolic disease and diabetes.
New York, New YorkAges 16–80 - NCT04869683Recruiting
Collecting blood and tissue samples in myelodysplastic syndrome
This trial collects and stores biological samples (like blood or other lab materials) from people with myelodysplastic syndrome, to help researchers learn more about the disease. It may help future diagnosis or treatments, even if it doesn’t directly test a new medicine.
BrestAges 18 years+ - NCT04494945Recruiting
Help for people with inherited cancer risk genes
This study helps identify people who may have an inherited cancer risk and checks how best to support them. Depending on the group, you may only fill out surveys, or you may also create an app account and possibly provide a DNA sample.
Portland, OregonAges 18 years+ - NCT04239157RecruitingPhase 2
Canakinumab shot for lower-risk MDS and related blood disorders
This trial tests canakinumab, a medication that blocks inflammation, given as a skin (subcutaneous) injection. It is for adults with lower-risk myelodysplastic syndromes (MDS) or a related condition (chronic myelomonocytic leukemia or CCUS) to see if it can improve blood counts and reduce problems like anemia or transfusion needs.
Houston, TexasAges 18 years+ - NCT04047641RecruitingPhase 1/Phase 2
Quizartinib with chemotherapy for new or returning leukemia
This trial tests a combination of anti-leukemia drugs (including quizartinib) together with chemotherapy for people with acute myeloid leukemia (AML), a rare mixed-type leukemia, or high-risk myelodysplastic syndrome (MDS). It may help by trying to better control cancer in people whose disease is newly diagnosed or has returned/not responded to prior treatment.
Houston, TexasAges 18 years+ - NCT03383575RecruitingPhase 2
Azacitidine plus enasidenib for IDH2-mutant MDS
This trial tests whether combining azacitidine with enasidenib helps people with myelodysplastic syndrome (MDS) that has an IDH2 mutation. It’s designed for people who are either new to “hypomethylating” treatment or whose MDS returned/worsened after it.
Baltimore, MarylandAges 12 years+ - NCT02958462Recruiting
Clinic study for early blood or bone marrow warning signs
This study follows people who may have early, unclear blood count problems or certain genetic/immune patterns that can later lead to bone marrow failure or blood cancers. It may help doctors better understand risks over time and guide earlier care.
Scottsdale, ArizonaAges 18 years+ - NCT01624805RecruitingPhase 2
Immunosuppressant treatment to help low blood counts in some bone marrow disorders
This trial tests a group of immune-suppressing medicines (with steroids and growth-factor injections) for people with aplastic anemia or certain low-risk myelodysplastic syndromes. The goal is to improve bone marrow function and reduce problems caused by low blood counts, such as fatigue, infections, or bleeding.
Houston, TexasAges 18 years+ - NCT02115295RecruitingPhase 2
Venetoclax plus chemotherapy for AML, high-risk MDS, or blast CML
This trial tests a combination treatment (venetoclax plus several chemotherapy medicines) for people with AML, high-risk MDS, or CML that has progressed into a blast phase. It may help by killing leukemia-like blood cells and improving disease control.
Houston, TexasAges 18–65 - NCT00499317Recruiting
Genetic study of chronic pelvic pain and bladder symptoms
This study looks for genetic (inherited) factors behind chronic pelvic pain and related urinary and sexual symptoms. You may be able to help researchers by providing a few samples and answering short questions, which could improve understanding of these conditions.
Boston, MassachusettsAges Any age - NCT00801489RecruitingPhase 2
New treatment for AML or high-risk MDS using combination chemo
This trial tests a combination treatment (chemo drugs plus a growth-factor medicine) for people newly diagnosed with acute myeloid leukemia (AML) or certain high-risk myelodysplastic syndromes (MDS). It may help by targeting cancer cells in the blood and bone marrow and improving remission chances.
Houston, TexasAges 18 years+ - NCT00768820RecruitingPhase 4
Study brain and mental health in people with 22q11 deletion
This trial looks at how thinking and mental health (psychiatric symptoms) show up in people who have a specific chromosome deletion. It may help researchers understand patterns in this condition and improve future care.
Petah TikvaAges Any age - NCT04704297RecruitingPhase 4
Trigger point shots for low back muscle pain
This trial tests whether injecting sore “trigger points” in low back muscles helps improve chronic low back pain. It compares treatments in people with specific low back muscle pain patterns and excludes several other causes of pain.
Tacoma, WashingtonAges 18 years+ - NCT01601171Recruiting
Genetic study of reproductive problems and cleft lip or palate
This study looks at genetic (inherited) causes of certain hormone and reproductive conditions, including Kallmann syndrome and cleft lip/palate. You might be asked for medical and family history and possibly a DNA sample, helping researchers find what drives these conditions.
Lausanne, Canton of VaudAges Any age - NCT04095195Recruiting
Pancreatic cancer risk registry for high-risk families
This study builds a registry of people with a higher-than-average risk of pancreatic cancer, based on family history or inherited gene conditions. If you join the extra imaging follow-up, it helps your care team monitor risk earlier.
Rozzano, MilanAges 18–80 - NCT03209414Recruiting
Study of frailty in heart patients receiving heart care
This trial looks at “frailty,” meaning reduced strength and resilience, in older adults with coronary artery disease who are getting heart care. It may help doctors better understand which patients are more at risk and how care decisions could be improved.
Zabrze, Upper SilesiaAges 65 years+ - NCT05786768RecruitingPhase 2/Phase 3
Obinutuzumab vs rituximab for relapsing childhood kidney syndrome
This study compares two antibody medicines, obinutuzumab and rituximab, to prevent relapses of steroid-dependent or frequently relapsing nephrotic syndrome in children. It looks at whether one option works better and is safer, including infection risk.
ParisAges 3–18 - NCT05938439Recruiting
Testing a magnetic device for Candy Cane Syndrome
This trial studies how well a small magnetic device works to relieve problems caused by Candy Cane Syndrome, such as pain, nausea, vomiting, or reflux-like symptoms. It will also check whether the device is safe for your anatomy and overall health.
BrusselsAges 18–85 - NCT06162546Recruiting
Registry for resistant nephrotic syndrome in children
This study tracks children with nephrotic syndrome whose kidney disease does not respond to usual treatments. It aims to collect information and samples to better understand resistant forms of the condition.
ViennaAges Up to 75 years - NCT06255678Recruiting
Angio-based check of heart artery treatment effect
This trial tests if special software can check the result of a balloon or stent procedure by looking at the X-ray movie of your heart arteries. It may help doctors decide if more treatment is needed.
WarsawAges 18 years+ - NCT05761314Recruiting
Study treatments for cancers in people with RASopathy
This study looks at people who have a RASopathy—a genetic condition—and also have solid tumors (tumor growths not in blood). To join, your RASopathy must be confirmed by genetic testing, not only by symptoms.
RomaAges Any age - NCT06353620Recruiting
Brain connections study for certain epilepsies and genetic syndromes
This study uses MRI and a special EEG to understand brain connections in people with specific types of epilepsy or genetic conditions like Angelman syndrome and Dup15q. It may help find better ways to treat these conditions.
Conegliano, TrevisoAges 6–75
Hear when a new Down Syndrome trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.
Common questions
- Are there clinical trials for down syndrome?
- Yes. Clin2 currently lists 3,048 recruiting down syndrome studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a down syndrome trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a down syndrome trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.