Clinical trials
Down Syndrome clinical trials
Below are recruiting down syndrome clinical trials, each written for real people, not researchers. We’re tracking 3,055 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05047354Recruiting
Study cholesterol metabolism in rare genetic conditions
This study looks at body chemistry (cholesterol-related lab findings) and physical features in people with Smith-Lemli-Opitz syndrome and related rare genetic cholesterol disorders. It may help researchers better understand these conditions and how they affect the body.
Bethesda, MarylandAges 1 day–100 years - NCT04900493Recruiting
Rett syndrome global patient and family registry
This registry collects health and family information about people with Rett syndrome (or specific changes in the MECP2 gene). It helps researchers understand the condition better and plan future studies. Participation may include sharing information for living individuals or those who have passed away.
Trumbull, ConnecticutAges Any age - NCT04923477Recruiting
Exercise study for shoulder tendon pain and pinching
This trial studies a specific therapeutic exercise program for rotator cuff tendon pain and subacromial pain (a pinching-type shoulder problem). It also looks at what changes in your symptoms and body function after the exercises.
Los Angeles, CaliforniaAges 18–55 - NCT04726241RecruitingPhase 1/Phase 2
Testing blood and bone marrow samples in young leukemia patients
This trial looks at blood and bone marrow samples from children and teens whose leukemia has come back or is hard to treat. The goal is to better understand the disease and guide future options for these patients.
Birmingham, AlabamaAges Up to 22 years - NCT04571658Recruiting
NEPTUNE Match study linking you to possible kidney research trials
This study is an add-on process that uses information from the NEPTUNE kidney study to see whether you might be able to join other kidney research trials. It may help connect eligible people with appropriate new treatment studies when they become available.
Atlanta, GeorgiaAges 1–80 - NCT04375631RecruitingPhase 1
Study of chemo plus donor stem cell transplant for blood cancers
This Phase 1 trial tests a specific chemotherapy plan followed by a reduced-intensity donor stem cell transplant for people with relapsed or treatment-resistant acute myeloid leukemia (AML), certain myelodysplastic syndromes (MDS), or chronic myelomonocytic leukemia (CMML). It may help by using donor stem cells to rebuild the bone marrow after intensive cancer treatment, in people who are not responding well to standard therapies.
Seattle, WashingtonAges 18 years+ - NCT04179981Recruiting
CPAP treatment effects in older COPD and sleep apnea patients
This study looks at whether using positive airway pressure (often called CPAP) improves health outcomes in older adults who have both COPD and obstructive sleep apnea. It may help find ways to reduce symptoms and complications from “overlap” breathing problems.
Detroit, MichiganAges 60 years+ - NCT04207580Recruiting
Follow-up study for children with newly diagnosed nephrotic syndrome
This study follows children in France who developed idiopathic nephrotic syndrome after 2018, to learn how the condition behaves over time. It may help doctors improve future care by tracking symptoms and outcomes.
AgenAges Up to 18 years - NCT03896269RecruitingPhase 1
CPX-351 for high-risk MDS or CMML that came back
This Phase 1 trial studies CPX-351, a cancer medicine, in people with high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) whose disease relapsed or did not respond to prior treatment. It may help shrink or control the leukemia and improve blood counts in patients who cannot or do not want a stem cell transplant.
Houston, TexasAges 18 years+ - NCT03775954Recruiting
Fetal heart testing in high-risk pregnancies after baby loss
This study checks unborn babies’ heart signals using special scans during pregnancy and then checks the baby’s heart shortly after birth. It may help researchers understand why certain high-risk pregnancies can lead to fetal demise and improve care.
Madison, WisconsinAges 18 years+ - NCT03661307RecruitingPhase 1/Phase 2
Study of quizartinib, decitabine, and venetoclax for acute myeloid leukemia or high-risk myelodysplastic syndrome
This trial combines three drugs to treat certain types of leukemia or a related blood condition. It may be an option for people who cannot have standard intensive chemo, or for those whose disease has come back.
Houston, TexasAges 18 years+ - NCT03670966RecruitingPhase 1/Phase 2
Targeted iodine therapy, then donor transplant for hard leukemias
This trial tests an antibody-linked radioactive iodine treatment, followed by a donor stem cell transplant, for people with relapsed or refractory high-risk acute leukemia or certain MDS. It may help patients by targeting leukemia cells and then using a new immune system to control the disease.
Seattle, WashingtonAges 18–75 - NCT03326921RecruitingPhase 1
T cell treatment for leukemia after a bone marrow transplant
This early-phase trial tests an immune “T cell” therapy made to recognize a specific leukemia target. It is for people whose acute leukemia has come back (relapsed) or did not fully respond after receiving a donor stem cell transplant.
Seattle, WashingtonAges Up to 80 years - NCT03128034RecruitingPhase 1/Phase 2
Astatine-211 treatment before stem cell transplant for high-risk blood cancers
This trial tests a targeted radiation drug (Astatine-211) given before a donor stem cell transplant for people with high-risk leukemia or certain bone-marrow cancers. The goal is to better control the cancer before the transplant.
Seattle, WashingtonAges 18–75 - NCT02725060Enrolling by invitation
Study of autoimmune changes in postural tachycardia syndrome
This trial studies whether an autoimmune (immune system–related) process is involved in postural tachycardia syndrome (POTS), a condition where your heart rate rises when you stand. The study may help researchers understand what causes POTS and how it could be treated or monitored.
Oklahoma City, OklahomaAges 18–50 - NCT02413450Enrolling by invitation
Using patient DNA cells to study inherited heart rhythm risks
This study uses your blood or genetic information to make “iPS cells,” lab-grown cells that can help researchers study inherited heart rhythm problems like LQTS, Brugada syndrome, CPVT, or early repolarization syndrome. It may help improve understanding of why these rhythms happen and guide future treatments.
Baltimore, MarylandAges 18–85 - NCT02399527Recruiting
Lymphatic problem registry for tracking outcomes
This study enrolls people with certain lymphatic (lymph fluid) conditions to collect information and track how they do over time. It may help doctors better understand outcomes and improve care.
Boston, MassachusettsAges Any age - NCT00697411Recruiting
Study of Aicardi syndrome in X-linked disorders
This trial looks at people with features that suggest Aicardi syndrome, a rare condition that can affect the brain and eyes and may include seizures. Researchers want to better understand the condition and how it shows up, which may help guide care in the future.
Houston, TexasAges Any age - NCT00556530Recruiting
Study genes and severity in 22q11.2 deletion
This study looks at how genetic differences affect how severe 22q11.2 deletion syndrome is for different people. It may help researchers understand what drives symptom severity and guide future care.
New York, New YorkAges Any age - NCT06782373RecruitingPhase 2
Pacritinib for VEXAS syndrome
This trial tests whether the drug pacritinib can safely reduce inflammation and improve symptoms in people with VEXAS syndrome, a rare genetic condition that causes inflammation in various parts of the body. It may be a new treatment option for those who still have inflammation despite taking steroids.
Scottsdale, ArizonaAges 18 years+ - NCT07226726RecruitingPhase 1
Stem cell exosome treatment for congenital myasthenic syndrome
This trial tests a treatment made from stem cell exosomes (tiny particles that may help repair cells) for people with congenital myasthenic syndrome, a muscle-weakening condition. It aims to see if this treatment is safe and could improve symptoms.
Naples, FloridaAges 18 years+ - NCT07298603Enrolling by invitation
Nutrition plan for women with PCOS
This study tests whether a specific nutrition plan can help women with PCOS improve their health. You would follow a guided diet and have check-ins to see how it affects your symptoms and well-being.
Samsun, AtakumAges 20–45 - NCT06303193RecruitingPhase 1/Phase 2
Testing a kinase inhibitor drug for MDS and MDS/MPN in adults and teens
This trial tests a drug called pacritinib in people with myelodysplastic syndromes (MDS) or MDS/myeloproliferative neoplasms (MDS/MPN). The drug targets several proteins that may help control the disease, especially for those who have not responded to other treatments.
Bethesda, MarylandAges 12–120 - NCT07786415Recruiting
Posture and physical function in college students with upper cross syndrome
This study looks at how posture affects everyday function in people with a common muscle imbalance called upper cross syndrome. It may help you understand your condition better, but you must be a college student in a specific age range and have this posture issue.
CairoAges 20–29
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Common questions
- Are there clinical trials for down syndrome?
- Yes. Clin2 currently lists 3,055 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.